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HAPSCD: Bone Marrow Transplant for Sickle Cell Disease

About this study 

Sickle cell disease is a serious blood disorder. It can cause severe pain, organ damage, and other health problems. Some medicines and blood transfusions can help control symptoms, but they do not cure the disease. A bone marrow transplant (also called a stem cell transplant) can cure sickle cell disease. It replaces unhealthy (sickle) blood-forming cells with healthy ones. Finding the right bone marrow donor with HLA typing is an important step for the transplant to be successful. But many patients do not have fully matched donors.  

This study is looking for a way to help more children and young adults get transplants. It uses stem cells from a family member who is at least a half-matched (haploidentical) donor. Stem cells are special cells that make new blood cells. Most stem cells are found in the bone marrow. 

Researchers are also studying a medicine called abatacept. It may help lower the risk of graft-versus-host disease. This is a serious problem that can happen after a transplant when donor cells attack the patient's body. 

Before the transplant, patients get medicines, chemotherapy, and a low dose of radiation to prepare their bodies. They then receive healthy stem cells from a family donor. After the transplant, the patients get abatacept and other medicines. These medicines help the body accept the new cells, which may lower the risk of side effects including graft-versus-host disease. Doctors will watch closely to see if the donor cells grow and make healthy blood cells. 

Patients stay in the hospital for 4–8 weeks. They also have regular checkups and tests for about 3–4 months after they go home. Doctors will look for side effects and check how well the transplant is working. Patients will be monitored for at least 3 years after their transplant. 

Researchers need to learn if this transplant approach is safe, what side effects occur, and if it can cure severe sickle cell disease without complications in children and young adults. They also need to learn if abatacept can help prevent graft-versus-host disease effectively while allowing the new donor cells to grow and work as they should. What researchers learn may help more children and young adults with sickle cell disease in the future. 

Eligibility overview 

  • Has severe sickle cell disease  
  • Does not have a fully-matched sibling donor 
  • Has a family donor who is a half-matched donor (haploidentical donor) 

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The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.

Overview

Full title:

HAPSCD: Haploidentical Donor Hematopoietic Cell Transplantation for Sickle Cell Disease

Study goal:

To study whether a haploidentical stem cell transplant and abatacept are safe and effective for severe sickle cell disease

Diagnosis:

Sickle cell disease

Age:

0–22 years

Clinical trial categories:

Sickle Cell Disease

For physicians and researchers

Patients accepted to St. Jude must be referred by a physician or other qualified medical professional. Learn how St. Jude can partner with you to care for your patient.

 

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