How Is Juvenile Arthritis Treated? A Parent’s Guide to Medications, Injections, and Eye Care

Key Takeaways:

  • Treatment starts early and aims for inactive disease. The goal of juvenile idiopathic arthritis (JIA) treatment is to stop inflammation before it damages joints, not just to manage pain, and pediatric rheumatologists now escalate treatment faster than they did a decade ago.
  • The main tools are joint injections, methotrexate, and biologics. Anti-inflammatory medicines like ibuprofen help with symptoms, but the medications that change the course of the disease are the ones that calm the immune system directly.
  • Oral steroids are used sparingly. Current guidelines favor targeted treatments over long courses of prednisone, which carry more side effects in growing children.
  • Eye exams are part of arthritis care. Uveitis, inflammation inside the eye, often has no symptoms in children with JIA and can only be caught through regular exams with an ophthalmologist.
  • Most children do well. With consistent treatment and monitoring, many children reach inactive disease, and a meaningful number are eventually able to taper off medication under their rheumatologist’s guidance.

Juvenile idiopathic arthritis (JIA)  is treated with a combination of medications that reduce inflammation and calm the overactive immune response that causes it, along with physical therapy, regular eye screening, and ongoing monitoring by a pediatric rheumatologist. Treatment is matched to the type of JIA a child has, how many joints are involved, and how the child responds, and it is adjusted over time with the goal of reaching inactive disease and keeping it there.

If you have recently learned that your child has JIA, you probably already know what the condition is. Our earlier post on when joint pain in children is more than growing pains covers how it is recognized and diagnosed. This one picks up where that left off and answers the questions families tend to have next: what the medications are, how they work, what the side effects look like in practice, and what the next few years are likely to involve.

What Is the Goal of JIA Treatment?

The goal is inactive disease: no swollen joints, no active inflammation on exam or labs, and no symptoms that limit what a child can do. That is a higher bar than “the pain is better,” and it exists for a reason. Inflammation that continues at a low level can quietly damage cartilage and bone, affect how a joint grows, and in some types of JIA, affect the eyes or internal organs.

This is why the approach to treating JIA has shifted. The American College of Rheumatology’s most recent guidelines emphasize moving to disease-modifying treatment sooner and relying less on medications that only address symptoms. As one of the guideline authors put it when the update was released, pediatric rheumatologists now escalate disease-modifying treatment more rapidly than in the past.

What Medications Are Used to Treat Juvenile Arthritis?

Families usually encounter four categories of medication. Not every child needs all of them, and the order and combination depend on the type of JIA.

NSAIDs (nonsteroidal anti-inflammatory drugs). Medicines such as ibuprofen and naproxen reduce pain and stiffness. They are helpful for comfort, and they are often part of early treatment, but they do not change the underlying disease. Guidelines now recommend shorter courses, since NSAIDs taken for long periods can irritate the stomach and affect the kidneys.

Steroid joint injections. For a child with arthritis in one joint or a few joints, an injection of a long-acting corticosteroid directly into the joint is often strongly recommended as initial treatment. A single injection can calm a joint for months, sometimes longer, with very little of the medication reaching the rest of the body. Young children are usually given light sedation or a numbing agent so the procedure is not frightening.

Conventional DMARDs, usually methotrexate. DMARD stands for disease-modifying antirheumatic drug. Methotrexate is the one used most often in children, and it has been the backbone of JIA treatment for more than 30 years. It is given once a week, as a liquid or tablet by mouth or as a small injection under the skin, and it works by dialing down the immune activity that drives joint inflammation. It takes six to twelve weeks to reach full effect, so patience in the first few months is part of the process.

Biologics. These are newer medications made from proteins that block specific signals the immune system uses to create inflammation. Different biologics target different signals: TNF inhibitors such as adalimumab and etanercept are common for arthritis affecting the joints, while IL-1 and IL-6 inhibitors are the preferred first-line treatment for systemic JIA, the type that causes fevers and rash. Biologics are given by injection at home or by infusion in a clinic. They are typically added when methotrexate alone is not enough, or used first in situations where guidelines support it.

Oral steroids. Prednisone and similar medicines reduce inflammation quickly, which makes them useful for severe flares or while waiting for a slower medication to take effect. Current guidelines recommend against relying on them as a long-term treatment because of their effects on growth, bone density, weight, and mood in children. When they are needed, the aim is the lowest dose for the shortest time.

An illustration showing the difference between a healthy knee joint and the knee joint of a child with JIA

Is Methotrexate Safe for Children?

This is the question parents ask most, and it deserves a direct answer. Methotrexate is used at much lower doses for arthritis than for cancer, and its safety record in children with JIA is well studied. A meta-analysis of 26 studies covering nearly 4,000 patient-years of treatment found that the most common side effects were mild, mostly nausea and temporary elevations in liver enzymes, that serious adverse events occurred in roughly 1 in 200 patients per year of treatment, and that more than two-thirds of children had no side effects at all over a year.

In practice, the side effects families notice are nausea on the day of the dose, fatigue, and occasionally mouth sores. Several things help: taking the dose at bedtime, taking a daily folic acid supplement, which is standard, and switching from oral to injectable methotrexate, which often reduces nausea. Regular blood tests check liver function and blood counts, and at Cure 4 The Kids Foundation those labs are drawn on site, usually at the same visit.

Anyone receiving methotrexate should avoid alcohol, and the medication is not safe during pregnancy. Your rheumatologist will raise it at the appropriate time.

What Are Biologics, and How Are They Given?

Biologics have changed what is possible for children with JIA. Before they became available, children whose arthritis did not respond to methotrexate had few good options. Today, a child whose disease is not controlled on one medication has several more to try.

How a biologic is given depends on the drug. Some are injected under the skin at home, every week or every other week, using a prefilled pen similar to an insulin pen. Others are given by intravenous infusion, which takes one to several hours and is repeated every few weeks. C4K has an on-site infusion suite designed for children, so families receiving infusion-based medications do not need to go to a hospital or a separate infusion center for each dose.

Because biologics and methotrexate work by reducing immune activity, children taking them are somewhat more likely to catch infections and may take longer to clear them. The practical rules are simple: call the care team about fevers, keep up with routine vaccines, and let the team know before any planned surgery or dental procedure, since medications are sometimes paused briefly around those events.

A young girl receiving a vaccine

Can My Child Get Vaccines While on Treatment?

Yes, with one category of exception. The ACR guidelines strongly recommend routine vaccinations for children with JIA, and the evidence does not support the common worry that vaccines trigger flares. Annual flu shots are encouraged. The European pediatric rheumatology vaccination recommendations, updated in 2021, reach the same conclusion: non-live vaccines are safe during immunosuppressive treatment and generally still work, though the response may be weaker during high-dose steroid treatment.

The exception is live vaccines, such as MMR and varicella (chickenpox), which are generally avoided while a child is immunosuppressed. Whenever possible, rheumatologists try to bring a child’s live vaccines up to date before starting methotrexate or a biologic. If that is not possible because treatment cannot wait, the team will map out a plan. Bring your child’s immunization record to the first rheumatology visit so this can be sorted out early.

Why Does My Child with Arthritis Need Regular Eye Exams?

Uveitis, inflammation of the middle layer of the eye, affects a meaningful share of children with JIA, most often those with the oligoarticular type, those diagnosed at a young age, and those whose blood tests show a positive ANA. The reason it requires a dedicated screening schedule is that in children it is usually silent. There is often no redness, pain, or obvious vision change until damage has already occurred. Left untreated, uveitis can cause cataracts, glaucoma, and permanent vision loss.

The 2019 ACR/Arthritis Foundation guideline on JIA-associated uveitis recommends regular exams with an ophthalmologist using a slit lamp, with the frequency set by the child’s risk level. Children at highest risk are seen as often as every three months. When uveitis is found, treatment starts with steroid eye drops, and if the inflammation does not settle within about three months, methotrexate or a TNF inhibitor such as adalimumab is added. Many children with uveitis are already on one of those medications for their arthritis, which is one reason good control of the joints and the eyes tends to go together.

Our rheumatology team coordinates ophthalmology referrals and keeps track of the screening schedule, so families have one person to call when a question comes up.

Pediatric rheumatologist helping a patient

What Else is Part of Treatment Besides Medication?

Medication controls the inflammation, but a child with arthritis also needs to keep moving, keep up in school, and feel like a kid. The ACR guidelines specifically recommend physical and occupational therapy to maintain range of motion and strength, prevent injury, and support every day and recreational activity. Rest is appropriate during a flare, but prolonged inactivity makes stiffness worse, and most children with well-controlled JIA can participate in sports and play with few limits.

How Long Will My Child Need Treatment?

There is no single answer, and your rheumatologist will be honest about that. JIA is a chronic condition, and some children need treatment through adolescence and into adulthood. Others reach inactive disease and stay there, and after a sustained period of remission on medication, typically a year or more, the care team may begin tapering. Current guidelines support reducing and stopping steroids once disease is inactive and, more cautiously, tapering biologics as well.

Tapering is done gradually, with monitoring, because arthritis can return when medication is reduced. That is not a failure; it is information, and it usually means restarting the medication that worked. The important thing is that stopping treatment is a decision made with the rheumatologist, never on a family’s own because a child seems fine. Inactive disease on medication and inactive disease off medication are two different things, and only the second one is confirmed by watching carefully after a taper.

Pediatric Rheumatologist at Cure 4 The Kids Foundation, Luciana Brandao Paim Marques
Dr. Paim, Pediatric Rheumatologist at Cure 4 The Kids Foundation

What Should Families Expect From the Pediatric Rheumatology Program at C4K?

The Pediatric Rheumatology Program at Cure 4 The Kids Foundation is led by Dr. Luciana Paim Marques, one of just three pediatric rheumatologists currently practicing in Nevada, working alongside nurse practitioner Angela Berg and a dedicated rheumatology nurse who handles coordination and monitoring between visits. The program treats JIA along with childhood lupus, dermatomyositis, vasculitis, scleroderma, autoinflammatory syndromes, and other connective tissue diseases.

Everything described in this post is available in one place: steroid joint injections in a child-focused setting, on-site labs, the infusion suite for IV medications, an on-site pharmacy for when a child’s situation calls for it, and more. Families are referred by their pediatrician or can contact C4K directly at (702) 732-1493.

Frequently Asked Questions About Juvenile Arthritis Treatment

Some children do reach lasting remission, and others have a condition that continues into adulthood. Early, consistent treatment gives the best chance of inactive disease and protects joints in the meantime.

Methotrexate is used at much higher doses to treat some cancers. For arthritis it is used at a small fraction of that dose, once a week, and it has been studied extensively in children with JIA. The side effects at arthritis doses are usually mild.

In most cases, yes, and activity is encouraged. Your rheumatologist and physical therapist can advise on any temporary limits during a flare or after a joint injection.

Children on methotrexate or biologics typically have labs every few months to monitor liver function, blood counts, and inflammation markers. At C4K these are drawn on site.

Biologics have been used in children with JIA for more than 20 years and are well studied. The main risk is a higher chance of infection, which is managed through monitoring, prompt attention to fevers, and keeping vaccines current.

Call the care team. For most JIA medications, a missed weekly dose is not an emergency, but the right way to handle it depends on the medication and how late the dose is.

Yes. Uveitis in children with JIA usually has no symptoms until it has caused damage, which is exactly why screening exams are scheduled regardless of how the eyes look or feel.

About the Author: Annette Logan-Parker brings over 30 years of experience in pediatric oncology and rare disease to her role as Founder and Chief Advocacy & Innovation Officer at Cure 4 The Kids Foundation. She has dedicated her career to improving outcomes for children with cancer and rare diseases, including ensuring equitable access to cutting-edge treatments for all families.

Understanding Blood Disorders in Children: What Parents Should Know About Pediatric Hematology

Key Takeaways:

  • Pediatric hematology is the medical specialty focused on blood disorders in children. These conditions affect red blood cells, white blood cells, platelets, and clotting factors, and they can range from mild and manageable to complex and lifelong.
  • Common childhood blood disorders include anemia, sickle cell disease, hemophilia, and thrombocytopenia. Each condition has distinct symptoms, but many share warning signs like unusual bruising, fatigue, and frequent infections.
  • Early diagnosis makes a meaningful difference. Many blood disorders in children are identifiable through routine blood work, newborn screening, or specialized laboratory testing.
  • Treatment is highly individualized. Depending on the condition, care may include nutritional support, medication, transfusions, or advanced therapies like hydroxyurea or stem cell transplant.
  • A pediatric hematologist provides specialized expertise that general pediatricians may not. Children’s blood and clotting systems differ from adults’, and accurate diagnosis requires providers trained specifically in pediatric blood disorders.

Pediatric hematology is the branch of medicine dedicated to diagnosing, treating, and managing blood disorders in infants, children, and adolescents. These disorders affect how the body produces blood cells, carries oxygen, fights infection, and controls bleeding. Because children’s bodies are still developing, blood disorders can influence growth, immunity, and overall health in ways that differ significantly from adults. A pediatric hematologist is a physician with specialized training in identifying and treating these conditions using age-appropriate diagnostic tools and therapies.

For many parents, hearing the words “blood disorder” can feel overwhelming. The good news is that pediatric hematology has advanced substantially over the past several decades. Conditions that were once poorly understood now have well-established treatment protocols, and children with blood disorders are living longer, healthier lives than ever before. This guide walks through the most common blood disorders seen in children, what symptoms to watch for, how these conditions are diagnosed, and what treatment may look like.

What Are the Most Common Blood Disorders in Children?

Blood disorders in children fall into several broad categories based on which component of the blood is affected. Some are inherited (passed down through a parent’s genes), while others are acquired through immune system changes, nutritional deficiencies, or other medical conditions.

The most common pediatric blood disorders include anemia (low red blood cells or hemoglobin), sickle cell disease, hemophilia and other bleeding disorders, thrombocytopenia (low platelet count), and clotting disorders (thrombophilia). Each of these conditions affects the blood in a different way, but they share one important feature: early recognition and specialized care can significantly improve a child’s quality of life.

Child with a bleeding disorder being supported by her pediatric care team

What Is Anemia in Children and What Causes It?

Anemia occurs when a child’s body does not have enough healthy red blood cells to carry adequate oxygen to tissues and organs. It is one of the most frequently diagnosed blood conditions in pediatric patients.

Several types of anemia affect children. Iron-deficiency anemia is the most common, often resulting from dietary gaps or periods of rapid growth. Aplastic anemia is a rarer and more serious form in which the bone marrow does not produce enough blood cells. Hemolytic anemia occurs when red blood cells are destroyed faster than the body can replace them. Thalassemia is an inherited condition that affects hemoglobin production and varies in severity.

Parents may notice symptoms such as persistent fatigue or low energy, pale skin (especially around the eyes, nails, and lips), irritability or difficulty concentrating, shortness of breath during physical activity, and a rapid heartbeat.

A complete blood count (CBC) is typically the first step in diagnosing anemia. Depending on the type, treatment may include iron supplementation, dietary changes, blood transfusions, or, in severe cases, referral for bone marrow evaluation.

Cellular-level view of sickle cells

How Does Sickle Cell Disease Affect Children?

Sickle cell disease (SCD) is an inherited blood disorder in which the body produces an abnormal form of hemoglobin, the protein inside red blood cells that carries oxygen. This abnormal hemoglobin causes red blood cells to become rigid, sticky, and shaped like a crescent or sickle. These misshapen cells can block blood flow through small vessels, leading to pain, organ damage, and increased risk of infection.

SCD is typically identified through newborn screening shortly after birth. A follow-up test called hemoglobin electrophoresis confirms the diagnosis. Children inherit sickle cell disease when they receive a sickle cell gene from each parent. A child who inherits only one copy of the gene has sickle cell trait, which is not a form of the disease, though they can pass the gene to their own children.

Symptoms can appear as early as four months of age and may include pain episodes (often called pain crises) in the chest, abdomen, joints, or limbs, swelling of the hands and feet (dactylitis), frequent infections, fatigue and jaundice (yellowing of the skin or eyes), and delayed growth.

Treatment for sickle cell disease is lifelong and focuses on preventing physical and behavioral complications, managing pain, and reducing the frequency of crises. Common approaches include hydroxyurea (a daily medication that helps reduce painful episodes), regular monitoring by a pediatric hematologist, preventive antibiotics and vaccinations, blood transfusions for severe anemia or stroke prevention, and hydration and pain management plans. Stem cell transplant (bone marrow transplant) remains the only established cure for sickle cell disease, though newer gene therapies are also emerging as potential options.

What Are Hemophilia and Other Bleeding Disorders in Children?

Hemophilia is an inherited bleeding disorder caused by a deficiency in one of the proteins (called clotting factors) that help blood clot. Children with hemophilia bleed longer than normal after an injury, surgery, or even without an obvious cause. There are two primary types: Hemophilia A, caused by a deficiency of clotting factor VIII, is the more common form. Hemophilia B, caused by a deficiency of clotting factor IX, is sometimes called Christmas disease.

Von Willebrand disease is another common bleeding disorder in children. It results from a deficiency or dysfunction of von Willebrand factor, a protein that helps platelets stick together to form clots.

Warning signs that a child may have a bleeding disorder include easy or excessive bruising, prolonged bleeding from cuts, dental work, or nosebleeds, blood in the urine or stool, and in the case of hemophilia, bleeding into joints (which can cause swelling and pain).

Treatment for bleeding disorders depends on the specific condition and its severity. Factor replacement therapy is the standard treatment for hemophilia, delivering the missing clotting factor through infusion. Other medications, including antifibrinolytic drugs, can help stabilize clots. Families also work with their care team to develop preventive plans that reduce injury risk while allowing children to remain active.

What Is Thrombocytopenia in Children?

Thrombocytopenia is a condition in which a child has a lower-than-normal number of platelets, the blood cells responsible for clotting. When platelet counts are low, even minor bumps or cuts can cause disproportionate bleeding or bruising.

The most common form in children is immune thrombocytopenia (ITP), in which the immune system mistakenly attacks and destroys platelets. ITP often develops after a viral illness and, in many children, resolves on its own within several months. Other causes of low platelet counts can include bone marrow disorders, certain medications, or inherited conditions.

Symptoms parents may notice include unexplained bruising, tiny red or purple spots on the skin (called petechiae), bleeding gums, and nosebleeds that are difficult to stop.

Diagnosis typically involves a CBC and a review of the child’s medical history. In some cases, additional testing may be needed to rule out other underlying conditions. Treatment for ITP ranges from watchful waiting (when the condition is mild) to medications that help boost platelet production or modulate the immune response.

What Are Pediatric Clotting Disorders?

While bleeding disorders involve too little clotting, thrombophilia and other clotting disorders involve too much. Children with these conditions form blood clots more easily than normal, which can obstruct blood flow and damage organs.

Pediatric clotting disorders may be inherited (genetic thrombophilia) or acquired, sometimes developing after surgery, prolonged immobility, central line placement, or in association with other medical conditions. Though less common in children than in adults, clotting disorders do occur and require careful monitoring.

Signs that a child may have a clotting disorder include swelling, pain, or warmth in a limb (particularly one-sided), unexplained headaches or vision changes, shortness of breath or chest pain, and skin discoloration near the site of a clot.

Treatment typically involves anticoagulant medications (blood thinners), along with lifestyle modifications and ongoing monitoring to prevent recurrence. A pediatric hematologist works closely with the family to balance effective treatment with the realities of an active child’s life.

How Are Blood Disorders Diagnosed in Children?

Diagnosing blood disorders in children begins with recognizing symptoms and pursuing appropriate testing. Many conditions are first detected through routine blood work at a well-child visit or through newborn screening programs.

Key diagnostic tools in pediatric hematology include the complete blood count (CBC), which measures red blood cells, white blood cells, and platelets; hemoglobin electrophoresis, which identifies abnormal hemoglobin types; coagulation studies (PT and PTT), which evaluate how well the blood clots; peripheral blood smear, which allows a lab professional to examine blood cells under a microscope; and genetic testing, which can confirm inherited conditions.

Laboratory accuracy matters enormously in pediatric hematology. Blood values in children differ from adults and change as children grow, which means pediatric-specific reference ranges and experienced laboratory professionals are essential for accurate interpretation.

The laboratory teams whose precision behind the scenes directly supports every diagnosis a pediatric hematologist makes. At Cure 4 The Kids Foundation, our CAP-accredited laboratory meets the gold standard for quality, ensuring that the test results our physicians rely on are accurate, reliable, and timely.

Child talking to his doctor during a pediatric clinical trial

When Should a Parent See a Pediatric Hematologist?

A referral to a pediatric hematologist may be appropriate when a child shows persistent or unexplained bruising or bleeding, chronic fatigue, paleness, or shortness of breath that doesn’t improve with standard treatment, abnormal blood counts detected on routine lab work, a family history of a known blood disorder, or symptoms that suggest a clotting event.

Pediatric hematologists are specifically trained to evaluate blood disorders in children, whose physiology, blood cell development, and clotting systems are distinct from adults. This specialized training matters because a lab result that would be normal for an adult may be abnormal for a child at a particular age, and because treatment plans must account for a child’s growth, activity level, and developmental needs.

Where Can Families in Nevada Find Pediatric Hematology Care?

Cure 4 The Kids Foundation provides comprehensive pediatric hematology services as part of its multidisciplinary care model. As Nevada’s only dedicated nonprofit pediatric cancer and rare disease treatment center, C4K offers evaluation, diagnosis, and long-term management for a wide range of non-malignant blood disorders, including anemia, sickle cell disease and other hemoglobinopathies, hemophilia and bleeding disorders, thrombocytopenia, and clotting disorders.

C4K’s team of hematology providers brings specialized expertise from nationally recognized training programs. The organization also participates in clinical research and trials, giving Nevada families access to the latest evidence-based treatment protocols. On-site services include a CAP-accredited laboratory, infusion center, physical therapy, child life specialists, social work support, and an on-site learning center in collaboration with Clark County School District so that children can continue their education during treatment.

Frequently Asked Questions About Pediatric Hematology

Pediatric hematology is the medical specialty focused on diagnosing, treating, and managing blood disorders in infants, children, and adolescents. These disorders can affect red blood cells, white blood cells, platelets, and the proteins involved in blood clotting.

Common signs include unusual or easy bruising, prolonged bleeding from cuts or nosebleeds, persistent fatigue or paleness, frequent infections, unexplained pain in the limbs or abdomen, and tiny red or purple dots on the skin (petechiae). If you notice any of these symptoms, speak with your child’s pediatrician about whether a referral to a hematologist is appropriate.

Stem cell transplant (bone marrow transplant) is currently the only established cure for sickle cell disease. Gene therapies have received FDA approval in recent years and represent a promising new frontier. However, many children with sickle cell disease are successfully managed with medications like hydroxyurea, preventive care, and regular monitoring by a pediatric hematologist.

A pediatric hematologist has completed additional fellowship training specifically focused on blood disorders in children. This matters because children’s blood cell development, normal lab values, and clotting systems change as they grow. Pediatric hematologists are trained to interpret results and design treatment plans within these age-specific parameters.

Yes. Cure 4 The Kids Foundation offers comprehensive pediatric hematology services, including evaluation, diagnosis, and ongoing management of conditions such as anemia, sickle cell disease, hemophilia, thrombocytopenia, and clotting disorders. C4K’s team of hematology providers is supported by a CAP-accredited laboratory, infusion center, and wraparound support services.

About the Author: Annette Logan-Parker brings over 30 years of experience in pediatric oncology to her role as Founder and Chief Advocacy & Innovation Officer at Cure 4 The Kids Foundation. She has dedicated her career to improving outcomes for children with cancer and ensuring equitable access to cutting-edge treatments for all families.

Gene Therapy for Hemophilia B: One Infusion, No More Weekly Treatments

Key Takeaways:

  • Gene therapy for hemophilia B targets the root cause of the condition — the faulty gene — rather than replacing the missing clotting factor from outside the body. A single intravenous infusion delivers a working copy of the factor IX gene to the liver, enabling the body to produce its own clotting factor.
  • Cure 4 The Kids Foundation completed Nevada’s first Hemgenix infusion in 2024 and is one of only a handful of centers on the West Coast to have administered this treatment. To date, C4K has successfully completed three Hemgenix infusions.
  • Gene therapy is not a cure. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. Long-term monitoring is essential.
  • Hemgenix is currently approved for adults only (18 and older). Pediatric gene therapy trials are in development but not yet available for children.
  • New treatment options are expanding rapidly. Beyond gene therapy, the FDA approved fitusiran (Qfitlia) in 2025 — a subcutaneous injection given once every two months that works for both hemophilia A and B, approved for patients aged 12 and older.

Gene Therapy for Hemophilia B: A vial of blood for the Factor IX test

What Does Gene Therapy for Hemophilia Look Like in Practice?

Imagine living your entire life tethered to a treatment schedule. Every week — sometimes multiple times a week — you need an intravenous infusion of a clotting factor just to prevent a bleed that could send you to the hospital. You plan your work around it. Your vacations. Your daily routine. It has been this way since you were a child.

Now imagine a single infusion that could change all of that.

That’s not a hypothetical. It happened right here in Nevada. And it happened at C4K.

What is Gene Therapy for Hemophilia B?

Gene therapy for hemophilia B is a one-time intravenous treatment that delivers a working copy of the factor IX gene to the liver using a modified, harmless virus as a carrier. Once in place, the liver cells begin producing factor IX on their own — the clotting protein the body was never able to make before. Unlike traditional replacement therapy, which requires lifelong intravenous infusions, gene therapy addresses the genetic root cause of the bleeding disorder in a single treatment.

Why Was Nevada’s First Gene Therapy Infusion for Hemophilia a Milestone?

In 2024, Cure 4 The Kids Foundation completed Nevada’s first successful infusion of Hemgenix®, an FDA-approved gene therapy for adults with hemophilia B. C4K is one of only five centers on the West Coast that has administered this treatment — with just two additional centers currently trained to do so. Nationally, the number of sites approved to provide Hemgenix remains extremely limited, making access to this therapy a significant barrier for many patients across the country. To date, C4K has successfully completed three Hemgenix infusions.

The treatment was administered in our infusion suite under the supervision of Dr. Aimee Foord, director of Cure 4 The Kids Foundation’s Bleeding and Clotting Disorders Clinic, and Dr. Joseph Lasky, our medical director.

Our first patient was a 39-year-old man from Arizona who had lived with hemophilia B his entire life, requiring regular factor IX infusions to prevent dangerous bleeding episodes. After a single Hemgenix infusion, his body began producing its own factor IX.

More than a year later, he has maintained normal factor levels without a single additional infusion. Our subsequent patients have seen similarly promising results.

While this is a fantastic medical milestone, for this patient, it means so much more: It’s a fundamentally different life.

What Is Hemophilia, and Why Does It Require Lifelong Treatment?

To understand why gene therapy matters, it helps to understand what hemophilia is at its most basic level.

Hemophilia is a genetic bleeding disorder. People with hemophilia B are missing a working copy of the gene that tells the body how to make factor IX — a protein the blood needs to form clots. Without it, even a minor injury can lead to prolonged, sometimes dangerous bleeding. Internal bleeding, particularly around joints, can cause chronic pain and permanent damage over time.

For decades, the standard treatment has been replacement therapy: regular intravenous infusions of the missing clotting factor. It works, but it’s a lifelong commitment. Depending on severity, a patient may need infusions multiple times per week — every week — for the rest of their life.

How Does Hemgenix Gene Therapy Work?

Gene therapy takes a completely different approach. Instead of replacing the missing protein from outside the body, it addresses the root cause: the faulty gene itself.

Hemgenix works by delivering a functional copy of the factor IX gene directly to the patient’s liver cells using a modified, harmless virus as a carrier. Once the gene is in place, the liver cells begin producing factor IX on their own. The goal is for the body to take over — making the clotting factor it was never able to produce before.

It’s a single, one-time intravenous infusion. Not a daily medication. Not a weekly treatment. One infusion.

Is Gene Therapy a Cure for Hemophilia?

Gene therapy is a remarkable advancement, but it’s important to understand what it is and what it isn’t.

It is not a cure. Hemgenix does not fix or replace the original faulty gene. It adds a working copy. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. Long-term monitoring is essential.

It is currently approved for adults only. Hemgenix is approved for adults aged 18 and older with hemophilia B who use factor IX prophylaxis or who have a history of serious bleeding episodes. Pediatric gene therapy trials are in development, but this treatment is not yet available for children.

Results can vary. Five-year follow-up data from the Phase 3 HOPE-B study, published in the New England Journal of Medicine, showed that Hemgenix reduced adjusted annualized bleeding rates by 63% through five years post-infusion, with mean factor IX activity levels sustained at 36.1%. In the trial, 94% of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years. But individual responses differ, and ongoing research continues to track long-term outcomes.

It requires ongoing monitoring. After gene therapy, patients need regular follow-up to track factor levels, liver function, and overall response to treatment. At C4K, our team provides that long-term monitoring and support.

Where Is Gene Therapy for Bleeding Disorders Heading?

Hemgenix was the first gene therapy approved for hemophilia B (2022), and Roctavian became the first approved for hemophilia A in 2023. These approvals represented a new era in bleeding disorder treatment — one that targets the genetic root of these conditions rather than managing symptoms. (Note: BioMarin announced in early 2026 that it is withdrawing Roctavian from the market after commercial challenges. While this is a setback for hemophilia A gene therapy specifically, multiple next-generation gene therapies for hemophilia A are in active clinical development.)

The landscape is evolving rapidly. New therapies are also expanding options for patients. In March 2025, the FDA approved fitusiran (Qfitlia), a different kind of treatment that works by rebalancing the body’s clotting system rather than replacing the missing factor. It’s given as a subcutaneous injection once every two months and is approved for adults and pediatric patients aged 12 and older with hemophilia A or B — offering patients a less burdensome prophylactic option with as few as six injections per year.

Research is also underway to develop gene therapies for children, to improve the durability of factor expression over time, and to explore gene-based treatments for other bleeding disorders beyond hemophilia. The science is moving fast — and C4K is committed to staying at the leading edge of what’s available for our patients.

A patient at Cure 4 The Kids Foundation receiving an infusion in the organization's infusion suite

What Does a Life Without Weekly Infusions Look Like?

For someone who has never lived with a bleeding disorder, it’s hard to understand what weekly factor infusions mean in practice. It’s not just the time in a chair. It’s the planning. The anxiety. The constant awareness that without your next infusion, an ordinary injury could become an emergency.

For a child growing up with hemophilia, it shapes everything — what sports they play, whether they go on the school camping trip, how their parents navigate every bump and bruise with a calculation most families never have to make.

Gene therapy doesn’t erase the diagnosis. But for the patients it works for, it can remove the most burdensome part of living with it. And that changes a life.

How Can I Learn More About Gene Therapy for Hemophilia B?

If you or a family member is living with hemophilia B and you want to learn more about whether gene therapy may be an option, our Bleeding and Clotting Disorders Clinic is here to help. Dr. Aimee Foord and our hematology team can walk you through what’s available, what the process looks like, and what to expect.

To schedule a consultation, call us at (702) 732-1493.

At C4K, we believe every patient deserves access to the most advanced care available — right here in Nevada. No patient is ever turned away for financial reasons. That is our promise.

Frequently Asked Questions

No. Gene therapy for hemophilia B adds a working copy of the factor IX gene but does not fix or replace the original faulty gene. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. However, for many patients, gene therapy significantly reduces or eliminates the need for routine factor IX infusions.

Hemgenix is currently FDA-approved for adults aged 18 and older with hemophilia B who use factor IX prophylaxis therapy or who have a history of current or life-threatening bleeding episodes. Eligibility also depends on testing for factor IX inhibitors and liver health. Your hematologist can help determine if you’re a candidate.

Five-year follow-up data from the HOPE-B clinical trial showed that factor IX activity levels remained sustained at a mean of 36.1%, and 94% of patients remained free of continuous prophylaxis through four years. Research is ongoing to track outcomes beyond five years.

Cure 4 The Kids Foundation in Las Vegas is the only center in Nevada to have administered Hemgenix gene therapy for hemophilia B. C4K is one of a limited number of centers on the West Coast offering this treatment. To learn more, contact the Bleeding and Clotting Disorders Clinic at (702) 732-1493.

Not yet. Hemgenix is currently approved only for adults aged 18 and older. Pediatric gene therapy trials are in development, and C4K is committed to offering the latest treatments as they become available.

In March 2025, the FDA approved fitusiran (Qfitlia), a subcutaneous injection given once every two months for patients aged 12 and older with hemophilia A or B. Unlike gene therapy or factor replacement, Qfitlia works by rebalancing the body’s clotting system. Your hematologist can help you understand which treatment options are right for you.

About the Author: Annette Logan-Parker brings over 30 years of experience in pediatric oncology to her role as Founder and Chief Advocacy & Innovation Officer at Cure 4 The Kids Foundation. She has dedicated her career to improving outcomes for children with cancer and ensuring equitable access to cutting-edge treatments for all families.

She Wasn’t Clumsy. She Wasn’t Dramatic. She Had a Bleeding Disorder No One Tested For.

Key Takeaways:

  • Bleeding disorders in girls are common but commonly missed. Von Willebrand disease is the most common bleeding disorder, affecting boys and girls equally — but girls are far less likely to be diagnosed.
  • Girls wait an average of 16 years for a diagnosis. Heavy periods, easy bruising, and prolonged nosebleeds are often dismissed as normal rather than recognized as symptoms of a bleeding disorder.
  • An estimated 2 million women in the U.S. may have an undiagnosed bleeding disorder. Many families don’t realize their bleeding is abnormal because other women in the family share the same undiagnosed condition.
  • Early diagnosis changes everything. It makes surgeries safer, periods treatable, and protects against life-threatening complications during pregnancy and childbirth.
  • You are not overreacting. If your daughter’s symptoms concern you, ask your pediatrician for a referral to a hematologist. At C4K, no child is ever turned away for financial reasons.

Why Bleeding Disorders in Girls Go Undiagnosed — and What Parents Can Do

She bruised easily. Everyone said she was clumsy.

Her nosebleeds wouldn’t stop. Everyone said she’d grow out of it.

Her periods left her doubled over and anemic. Everyone said that’s “just how it is for some girls.”

She wasn’t clumsy. She wasn’t dramatic. She had von Willebrand disease. And no one tested her for 16 years.

If this sounds familiar — if you’re reading this and thinking about your own daughter, your niece, or even yourself — you’re not alone. And you’re not imagining it.

What Are Bleeding Disorders, and Why Are Girls More Likely to Be Missed?

Bleeding disorders are conditions that affect the blood’s ability to clot properly. They include hemophilia, von Willebrand disease, rare factor deficiencies, platelet disorders, and more. Every March, during Bleeding Disorders Awareness Month, the bleeding disorders community comes together to raise awareness about these conditions. Since 2016, when the U.S. Department of Health and Human Services expanded what was previously Hemophilia Awareness Month, March has recognized the full spectrum of inheritable bleeding disorders. The red tie is the symbol of this community, representing the blood ties that bind us together.

At C4K, our Bleeding and Clotting Disorders Clinic, led by Dr. Aimee Foord, treats patients of all ages across this full spectrum. We see firsthand what these conditions look like in children — and we see how often they go unrecognized, especially in girls.

Dr. Aimee Foord, Pediatric Hematologist/Oncologist at Cure 4 The Kids Foundation

Most people hear “bleeding disorder” and think of hemophilia in boys. But the most common bleeding disorder isn’t hemophilia — it’s von Willebrand disease, and it affects boys and girls equally. The difference? Girls are far less likely to be diagnosed.

Here’s why that matters:

Why Are Bleeding Disorders in Girls Dismissed as Normal?

Because girls’ bleeding symptoms look different — and they’re easier to dismiss.

When a boy with hemophilia bleeds into a joint, it’s visible and alarming. When a teenage girl soaks through a pad every hour, misses school every month, and is chronically exhausted, she’s told: “That’s just how it is for some women.” Or: “You’ll regulate eventually.” Or: “Take some ibuprofen.”

Research confirms what many families already feel: women with inherited bleeding disorders often wait well over a decade — about 14 years on average — for a diagnosis, compared to under 10 years for men, even when bleeding begins at similar ages. The problem isn’t that girls aren’t showing symptoms. It’s that their symptoms are being normalized.

There’s another layer to this that we see in our clinic: many families don’t recognize heavy periods as abnormal because other women in the family have the same experience, unknowingly sharing the same undiagnosed condition. Mothers, daughters, aunts, sisters. Generation after generation told their bleeding is normal.

As Dr. Nidhi Bhatt at St. Jude Children’s Research Hospital has noted, adolescents often don’t feel comfortable talking about their bleeding — and that silence is one of the major reasons heavy menstrual bleeding goes undetected. St. Jude created the EAGER Clinic (Empowering Adolescents with Gynecology and Hematology Resources and Care) to address exactly this gap — bringing hematology, gynecology, and nursing together for adolescent patients. Their data reflects what we see nationally: only 1 in 3 women with heavy menstrual bleeding ever seek care for it.

What Are the Signs of a Bleeding Disorder in Girls?

Every child is different, and heavy bleeding doesn’t automatically mean a bleeding disorder. But these are signs worth discussing with your pediatrician or a hematologist:

  • Periods that last longer than 7 days
  • Soaking through a pad or tampon in an hour or less
  • Passing blood clots larger than a quarter
  • Missing school, sports, or social activities because of her period
  • Chronic fatigue or a diagnosis of iron-deficiency anemia
  • Easy bruising or frequent nosebleeds
  • Excessive bleeding after dental work, surgery, or an injury
  • A family history of heavy bleeding, frequent nosebleeds, or hysterectomies due to heavy periods

That last one matters more than many people realize. Von Willebrand disease is inherited. If the women in your family have always had “bad periods,” it’s possible there’s an undiagnosed bleeding disorder running through the family line.

What Happens When a Bleeding Disorder Goes Undiagnosed?

Delayed diagnosis isn’t just frustrating. It causes real harm.

Women who experienced multiple bleeding events before their diagnosis were nearly three times more likely to undergo a hysterectomy — a surgery that, in many cases, could have been avoided with earlier treatment.

Studies show that 74% of women with von Willebrand disease reported excessive bleeding from multiple sites. Twenty-five percent had undergone a hysterectomy, compared to just 9% in control groups. And rates of depression and anxiety in patients with VWD exceed 60%.

For teenage girls, the impact shows up in missed school days, withdrawal from activities they love, chronic exhaustion, and the quiet erosion of confidence that comes from being told — over and over — that nothing is wrong when they know something is.

Symptoms of Von Willebrand Disease

How Does Early Diagnosis Change a Girl’s Life?

When we identify a bleeding disorder early, everything shifts.

Surgeries become safer because the care team knows what they’re working with. Injuries are managed appropriately. Heavy periods can be treated — not dismissed. And the young woman at the center of it finally has answers instead of doubt.

Early diagnosis also protects her future. Women with bleeding disorders face increased risks during pregnancy and childbirth. Knowing about a bleeding disorder before that moment — not during a hemorrhage in the delivery room — can be lifesaving.

Where Can Families Get Help for Bleeding Disorders in Nevada?

At Cure 4 The Kids Foundation, our Bleeding and Clotting Disorders Clinic provides comprehensive evaluation and treatment for children and adults with bleeding disorders. Dr. Aimee Foord and our hematology team specialize in the conditions that are most often missed — especially in girls and young women.

If your daughter’s symptoms sound like what we’ve described here, we want you to know: you are not overreacting. You are not imagining it. And you don’t have to figure it out alone.

To schedule an appointment or request a referral, call us at (702) 732-1493.

No child is ever turned away for financial reasons. That is our promise.

March is Bleeding Disorders Awareness Month

This Month and Beyond

Bleeding Disorders Awareness Month is about more than wearing a red tie — though we hope you’ll do that too. It’s about expanding who we think of when we think of bleeding disorders.

It’s the teenage girl who can’t make it through a school day. It’s the mother who assumes her heavy periods are normal because her own mother had the same thing. It’s the young woman heading into surgery without anyone thinking to check her clotting factors first.

They deserve answers. They deserve care. And at C4K, they’ll find both.

If you think your daughter might have a bleeding disorder, don’t wait. Talk to your pediatrician. Ask for a referral. Or call us directly.

Because she’s not clumsy. She’s not dramatic. And she deserves to know what’s really going on.

Frequently Asked Questions

Yes. As many as 1 in 10 women with heavy menstrual bleeding may have an underlying bleeding disorder such as von Willebrand disease. Signs include periods lasting longer than 7 days, soaking through a pad or tampon in an hour or less, passing large blood clots, chronic fatigue, and iron-deficiency anemia. If your daughter is missing school or activities because of her period, it’s worth discussing with a pediatrician or hematologist.

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting up to 1% of the population. It occurs when the blood lacks sufficient von Willebrand factor, a protein that helps blood clot. VWD affects boys and girls equally, but girls are often diagnosed later — or not at all — because their symptoms, particularly heavy periods, are frequently dismissed as normal.

Diagnosis typically involves blood tests that measure von Willebrand factor levels and clotting function. These are specific tests that your pediatrician can order or a hematologist can perform. Standard blood work such as a CBC does not test for von Willebrand disease, which is one reason it’s so often missed. If your daughter has symptoms, ask specifically for von Willebrand disease testing.

Hemophilia is one type of bleeding disorder, but it’s not the only one — and it’s not the most common. Von Willebrand disease, rare factor deficiencies, and platelet disorders are all part of the bleeding disorders spectrum. Hemophilia is more commonly associated with boys, which has contributed to the misconception that bleeding disorders don’t affect girls. They do.

Cure 4 The Kids Foundation’s Bleeding and Clotting Disorders Clinic, led by Dr. Aimee Foord, provides comprehensive evaluation and treatment for children and adults with bleeding disorders in Southern Nevada. No child is ever turned away for financial reasons. Call (702) 732-1493 or visit cure4thekids.org to schedule an appointment or request a referral.

About the Author: Annette Logan-Parker brings over 30 years of experience in pediatric oncology to her role as Founder and Chief Advocacy & Innovation Officer at Cure 4 The Kids Foundation. She has dedicated her career to improving outcomes for children with cancer and ensuring equitable access to cutting-edge treatments for all families.

Congratulations Dr. Lasky!

We’re proud to announce some news about Dr. Lasky!  He has been promoted to director of our Bleeding and Clotting Disorders Clinic.

As one of our board-certified Pediatric Hematology-Oncology physicians, he will continue to see patients affected by childhood cancer. But he will also oversee all aspects of medical treatment for our patients affected by bleeding and clotting disorders, such as hemophilia and von Willebrand disease and other related conditions.

Since 2009, Dr. Lasky has served as principal investigator for a number of clinical trials and has participated in other research activities aimed at improving treatments for those with bleeding and clotting issues.

Congratulations Dr. Lasky!

CSL627_3001 Hemophilia A

Bleeding and Clotting Disorders

Protocol:CSL627_3001

Condition or Disease: Hemophilia A

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII SingleChain, CSL627) in Subjects with Severe Hemophilia A

Principal I investigator: Joseph Lasky III, MD

ALN-AT3SC-003 Hemophilia A or B

Bleeding and Clotting Disorders

Protocol:ALN-AT3SC-003

Condition or Disease: Hemophilia A or B

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

Principal I investigator: Joseph Lasky III, MD

More Info: //clinicaltrials.gov/ct2/show/NCT03417102

ALN-AT3SC-004 Hemophilia A or B

Bleeding and Clotting Disorders

Protocol:ALN-AT3SC-004

Condition or Disease: Hemophilia A or B

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

Principal I investigator: Joseph Lasky III, MD

More Info: //clinicaltrials.gov/ct2/show/NCT03417245

1160.106 Venous thrombosis

Bleeding and Clotting Disorders

Protocol:1160.106

Condition or Disease: Venous thrombosis

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: Open-label, randomized, parallel-group, active-controlled, multi-centre, non-inferiority study of dabigatran etexilate versus standard of care for venous thromboembolism treatment in children from birth to less than 18 years of age: The DIVERSITY study

Principal I investigator: Alan Ikeda, MD

More Info: //clinicaltrials.gov/ct2/show/NCT01895777