Haploidentical Hematopoietic Stem Cell Transplantation

Korábbi kezelés

• Patients with uncontrolled bacterial, viral or fungal infections (undergoing appropriate treatment and with progression of clinical symptoms) within 1 month prior to conditioning. Patients with febrile illness or suspected minor infection should await clinical resolution prior to starting conditioning.
• Patients who have an intolerance to or have received alemtuzumab in the prior 6 months will be excluded from enrollment unless alemtuzumab is replaced with rabbit ATG in the conditioning regimen

Más betegség

• Patients seropositive for the human immunodeficiency virus (HIV)
• Patient with active Hepatitis B or C determined by serology and/or NAAT
• Active hepatitis, bridging fibrosis or cirrhosis on liver biopsy (biopsy required for patients on chronic transfusion therapy for > 1 year and evidence of iron overload with ferritin >1000 ng/mL)

Egyéb

• Life expectancy less than 6 months
• Pregnant or breastfeeding patients
• Patients with suitable 8/8 HLA matched related and unrelated donors

• First allogeneic transplant
• Age up to 22 years
• Patients with severe sickle cell disease (stroke, elevated TCD velocities, >2 acute chest syndrome, ongoing chronic red cell transfusion > 6 months)
• Patients with transfusion dependent thalassemia and evidence of iron overload
• Patients must have a related donor that is HLA-matched at >/=4 of 8 but <8/8 HLA-A, -B, -C and -DRB1
• Cardiac function: Shortening fraction >25%; ejection fraction >40%
• Estimated creatinine clearance greater than 50 mL/minute
• Pulmonary function: DLCO ≥40% (adjusted for hemoglobin) and FEV1≥50% in patients 7 years and older with normal cognitive function and able to perform the test adequately. If not able to complete the testing a CT chest will be required., oxygen saturation>91%
• Liver function: direct (conjugated) bilirubin < 2x the upper limit of normal and ALT/AST < 2.5x the upper normal limit.
• Signed informed consent.