A study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease using a Reduced-Intensity preparatory regime with Campath-1H Fludarabine and Melphalan
Investigation of Bone Marrow Transplantation for Non-Cancerous Diseases
Brief description of study.
The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation.
Detailed description of study
The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Sickle Cell Disease,Thalassemia
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Age: 1 years - 21 years
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Gender: All
Inclusion Criteria
Patients must be diagnosed with a non-malignant disorder benefitted by allogeniec stem cell transplantation (SCT)
Patients must have a related or unrelated donor (bone marrow or peripheral blood) matched at 7 or 8 of 8 HLA loci by molecular typing A, B, C, and DR) or a cord product matched at 5 -6 of 6 HLA loci (A, B and DRB1) by intermediate resolution typing at Class I and high resolution molecular typing at Class II loci.
Recipient age should be less than 21 years.
Medical criteria for exclusion of recipient: Karnofsky/Lansky performance status scale of <70; DLCO (corrected for hemoglobin) or pulmonary function tests <40% of expected values if performed; if PFTs not performed, O2 sats (<88%) at the time of enrollment or chest X ray that indicates evidence of active infection or a condition that markedly alters lung function as determined by study chair and local institutional PI. In the absence of PFTs, final eligibility determination will be the responsibility of the study chair/institutional PI LVEF (ejection fraction) <40% or LVSF (shortening fraction) <26%; serum creatinine >2.0 times normal for age; liver enzymes >5 times normal values; invasive infections meeting criteria in section 3.7; positivity for HIV I/II. If patient has Sickle Cell, Hgb S should be tested within 30 days of beginning conditioning and must be <30%.
Female patients of childbearing potential must have negative pregnancy test within 2 weeks of treatment and must not be lactating.
Written informed consent must be obtained from all recipients in accordance with the guidelines of the institution
This study investigates the use of bone marrow transplantation to treat diseases that are not cancer. Bone marrow transplantation involves replacing damaged or diseased bone marrow with healthy bone marrow stem cells. This is done to improve the function of the patient's blood and immune system.
Participants in this study will undergo a procedure that involves receiving stem cells from a donor. The donor's stem cells can come from bone marrow, peripheral blood, or cord blood. The study aims to evaluate how well this treatment works in improving the health of patients with specific non-cancerous diseases.
- Who can participate: Participants must be under 21 years old and have a non-cancerous disorder that benefits from stem cell transplantation. They must have a suitable donor and meet specific health criteria.
- Study details: Participants will receive stem cells from a donor, which can be from bone marrow, peripheral blood, or cord blood. The study will assess the effectiveness of this treatment in improving patient health.