A study of Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease using a Reduced-Intensity preparatory regime with Campath-1H Fludarabine and Melphalan

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: Hematopoietic Stem Cell Transplantation (HSCT) in Non-Malignant Disease | Sickle Cell Disease | Thalassemia
  • Age: Between 1 - 21 Years
  • Gender: Male or Female

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

Recruitment Status


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