In this study the investigators will prospectively investigate whether the addition of a 3-months long preconditioning with azathioprine to the alemtuzumab/TBI non-myeloablative conditioning results in improved disease-free survival and donor chimerism after allo-SCT in SCD patients. Furthermore, the investigators will evaluate whether azathioprine/hydroxyurea preconditioning leads to more patients being able to taper and discontinue sirolimus at 12 months post-transplantation.
| Erfan Nur, MD, PhD | |
| 0031-20 - 4442604 | |
| [email protected] |
| Management hematology | |
| 0031-20 - 4442604 | |
| [email protected] |
16 Years - 60 Years
Observational
All
Other
SCD patients with an HLA-identical matched sibling donor eligible for allogeneic stem cell transplantation.
Age 16 – 60 years
Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)
Patients and donors (MSD) must be able to sign consent forms for receiving and donating hematopoietic stem cells respectively. The sibling donor should be willing to donate.
Patients must be geographically accessible and willing to participate in all stages of treatment.
Eligible diagnoses: Patients with sickle cell disease such as sickle cell anemia (Hb SS), Hb/Sβ0-thalassemia, Hb/Sβ+-thalassemia, HbSC disease, HbSE disease, HbSD disease and Hemoglobin SO- Arab disease.
Poor performance status (ECOG>1).
Poor cardiac function: left ventricular ejection fraction<35%.
Poor pulmonary function: FEV1 and FVC<40% predicted.
Poor liver function: direct bilirubin >3.1 mg/dl
HIV-positive
Women of childbearing potential who currently are pregnant (Beta-HCG+) or who are not practicing adequate contraception.
Patients who have any debilitating medical or psychiatric illness that would preclude their giving informed consent or their receiving optimal treatment and follow-up. However, patients with history of stroke and significant cognitive deficit, that would preclude giving informed consent or assent will not be excluded, if they have a family member or significant other with Power of Attorney to also consent of their behalf.