Understanding How Patients Fare Years After Transplantation

Across North America, advances are being made in the use of blood and marrow transplants. For more than fifteen years, its applicability as a cure sickle cell disease continues to increase. As a result, there is a growing population of individuals who have been transplanted for sickle cell disease. Some who were children when they had this life saving therapy are now adults. STAR centers will analyze health information on approximately 200 persons who underwent transplant for sickle cell disease. For the first time, we will be able to study health outcomes and how people fared after five, ten, even fifteen years.

Your generous contribution to STAR will support this critical deepening of our understanding of the long-term health outcomes on children who had bone marrow transplants years ago.  Please donate now.

 

Reducing Graft Versus Host Disease (GVHD)

Graft Versus Host Disease can be a serious complication of blood and marrow transplantation.  It occurs when the immune cells in the donor graft (the blood or marrow transplant) recognize and react to differences in the host’s (the patient’s) body.  Using donors who are matched with the patient for HLA (human leukocyte antigen) proteins and administering immunosuppressive medications prevents graft versus host disease in most children. However,  some still develop chronic problems from GVHD like rash, diarrhea and mouth sores.  Very severe cases of GVHD can be fatal.

The likelihood of a child developing graft versus host disease varies depending on their age and their donor’s age.  The disease is more likely to occur in children who are ten and older and younger children whose donor is ten or older. Children who get a transplant from an unrelated donor are also more likely to get graft versus host disease. More effective medicines to prevent GVHD are needed for these children.  In this trial, we assess a new immunosuppressive medication, abatacept (Orencia®), which has shown promise at preventing GVHD in early trials involving patients transplanted for leukemia. This study is now open and actively recruiting patients.  Please click on the following link for more information: 

https://clinicaltrials.gov/ct2/show/NCT02867800

While Bone Marrow Transplant can cure sickle cell, GVHD can be a terrible complication.

Please support our work as we investigate how to reduce the likelihood of any child having to live with this crippling disease.

 

HLA Matched Related Stem Cell Transplantation for Children with Less Severe Sickle Cell Disease

Sickle cell disease deprives nearly all patients, even children with a relatively mild course, of a full life. A Human leukocyte antigen (HLA)[1] matched sibling donor transplant has been shown to be safe and effective for children with a history of severe complications. Complications of sickle cell disease include suffering from a stroke, recurrent acute chest syndrome, or frequent painful incidents of blocked blood vessels (vaso-occlusive crises). Because bone marrow transplant has risks, this therapy has historically been offered only to families of children with severe disease. Yet recent improvements in transplant for patients with severe disease, makes it possible to explore extending this therapy to those who are less affected by sickle cell disease, especially when there is an available HLA–matched sibling donor.

In this study, we hope to reduce the risk of known bone marrow transplant complications, such as graft-versus-host disease. For this reason, this study is limited to patients with less severe sickle cell disease who are under the age of 10 years and who have a HLA matched sibling donor also under age 10.

A reduced intensity chemotherapy-conditioning regimen will be given before transplant to prepare the patient to receive his/her sibling’s bone marrow cells. These medications have been shown to be safe and effective in a recent multicenter study for children with severe sickle cell disease. STAR investigators are hopeful that this proposed treatment could reduce the risk of known late effects of transplant and possibly preserve fertility. Patients will be monitored for up to five years after the transplant so we can better understand how this treatment impacts the brain, kidneys, and in females, ovarian function over time. We will use surveys to assess health issues to determine if transplant improves these patients’ quality of life.

The goals of this multicenter trial are to:

1. Assess the safety and value of using a reduced intensity conditioning regimen for HLA matched sibling transplants and

2. Address current gaps in our understanding of the long-term effects of transplants in children with sickle cell disease.

We hope to enroll 50 patients and 50 donors for this study. Participants will be followed at various times for 5 years.

Please support STAR as we explore ways to make the transplant process less challenging for those with a matched donor.

 

[1] Human leukocyte antigen (HLA) is a protein – or marker – found on most cells in the body. HLA is used to match the donor with the host. The best transplant outcomes happen when the patient’s HLA closely matches the donor’s HLA.