Cell Therapy and Bone Marrow Transplant Program
Hemoglobinopathies and Bone Marrow Transplantation

<p>Hemoglobinopathies and Bone Marrow Transplantation</p>

The Hemoglobinopathies Clinic within Texas Children’s Cell Therapy and Bone Marrow Transplant Program provides expert care for children with sickle cell disease, thalassemia, and other hemoglobin disorders. Our multidisciplinary team offers comprehensive evaluation, treatment, and long-term follow-up for children who may benefit from bone marrow transplant or cell therapy.

As part of one of the nation’s leading pediatric transplant programs, we perform more than 140 bone marrow transplants, and cell and gene therapy infusions each year. We combine specialized expertise, innovative therapies, and compassionate support to guide children with hemoglobinopathies and their families through every stage of care. Through coordinated, patient-centered care and clear communication, we strive to make the treatment journey as safe, seamless, and supportive as possible.

Why Choose Texas Children’s for Hemoglobin Disorders?

One Coordinated Care Team
Specialists in sickle cell care, bone marrow transplant, neurology, kidney, lung, heart, psychology, and social work collaborate as one team—so families receive clear guidance and seamless support at every step.

Expert Care with Access to New Therapies
Patients may have access to emerging treatments such as gene therapy and haploidentical transplantation, supported by participation in national research collaborations including the Sickle Cell Transplant Advocacy and Research Alliance (STAR). These partnerships help us continuously refine care and improve safety.

Care That Honors Every Family
We recognize that trust is built over time. Our team is experienced in caring for a culturally diverse community and is committed to respectful, transparent communication.

Support Beyond Transplant
Our survivorship program supports children as they return to school, reconnect with peers, and transition to adult care, while addressing emotional, social, and mental well-being.

Our program also works in close alignment with the National Marrow Donor Program (NMDP) to support donor access and referral pathways for children with hemoglobinopathies who lack matched donors.

Hemoglobinopathies We Treat

Our team provides transplant and cellular therapy for a wide range of hemoglobin disorders, including:

Sickle Cell Disease (HbSS, HbSC, HbSβ⁰, HbSβ⁺)
Thalassemia major and intermedia
Sickle/Thalassemia overlap syndromes
Transfusion-dependent hemoglobinopathies

Outcomes from our hemoglobinopathies transplant program are reported to and benchmarked through the Center for International Blood and Marrow Transplant Research (CIBMTR), supporting national research collaboration, outcomes analysis, and continuous quality improvement in pediatric hemoglobinopathies transplantation.

Explore Cell Therapy and BMT Resources

Treatment Options

We provide integrated, patient-centered care throughout the transplant journey, with treatment plans tailored to each child's unique needs. Our multidisciplinary team offers proactive monitoring and comprehensive support services, including neuroprotection, pulmonary and kidney care, infection prevention, fertility preservation, and mental health support.

We offer multiple transplant and curative therapy options based on each patient’s medical needs and goals, including:

Myeloablative Matched-Sibling Hematopoietic Stem Cell Transplant (HSCT)
Haploidentical (Half-Matched) HSCT using post-transplant cyclophosphamide (clinical trial coming soon)
Reduced-Intensity Conditioning Transplant for older or higher-risk patients using matched related or unrelated donors
Gene therapy for hemoglobinopathies

Meet our Team

Frequently Asked Questions

Is transplant or gene therapy right for my child (or for me)?

Not every patient needs transplant right away, and not every patient is the same. Our team carefully reviews your medical history, symptoms, and goals to help you understand whether transplant or another transformative therapy option may be right for you, now or in the future. The decision is always shared, thoughtful, and never rushed.

How safe is transplant today?

Transplant does carry risks, but it is safer today than ever before. At Texas Children’s, we use the latest approaches to reduce complications, including medicines that lower the risk of GVHD, close monitoring of the brain and organs, and access to advanced immune therapies if problems arise. Our team also works with national research groups to continually improve safety and outcomes.

What if we don’t have a matched sibling donor?

Many families worry about this, and you are not alone. In addition to matched sibling transplants, we offer half-matched (haploidentical) transplants, matched unrelated donor options, and gene therapy trials for eligible patients. We will review all possible options with you.

What will life be like during and after transplant?

Transplant is a journey that takes time. Most patients stay in the hospital for several weeks, followed by close follow-up after discharge. Our team supports not only medical recovery, but also emotional health, school planning, and returning to everyday life. Long-term follow-up helps protect health years after transplant.

How does your team support families emotionally and practically?

We know transplant affects the whole family. Our social workers, psychologists, and patient navigators help with emotional support, housing needs, school coordination, and connecting families to community and peer support. You are never expected to handle this alone.

To learn more about how we support families, click below:

Patient & Family Support

How do we get started or learn more?

The first step is a consultation either referred by your hematologist or requested by your family. This visit is focused on listening, education, and answering questions. Even if transplant is not the right choice right now, we are happy to help guide next steps and future planning.

Can we talk to another family who has been through transplant?

Yes. Through the National Marrow Donor Program (NMDP / Be The Match®) Peer Connect Program, families can choose to talk with trained peer volunteers who have personal experience with transplant either as patients, caregivers, or donors. If you’re interested, click on this link: https://www.nmdp.org/patients/transplant-support/patient-support-center/peer-connect

Learn More About the Cell Therapy & BMT Program

Care for hemoglobinopathies is one part of Texas Children’s broader program. To learn more about our complete transplant services, including cellular therapies, gene therapy, and transplantation for other conditions, visit the Cell Therapy and Bone Marrow Transplant Program page.