Haploidentical Hematopoietic Stem Cell Transplantation

Launched by CATHERINE BOLLARD · Jun 16, 2014

Keywords

ClinConnect Summary

Research subjects will undergo reduced intensity conditioning (Hydroxyurea, ATG, Fludarabine, Thiotepa, Melphalan) followed by infusion of a peripheral blood stem cell graft collected from haploidentical family donors that are CD34+ positively selected using the CliniMACS device. Sirolimus will be used for GVHD prophylaxis and given for 9 months post-transplant and then tapered off by one year

The use of the CliniMACS device for CD34 selection will be performed at CNMC through cross-reference of the master file for CliniMACS CD34+ Reagent by Milteyni Biotech (BB-MF 8061).

CliniMACs is an ...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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.
• Exclusion Criteria:
• • Life expectancy less than 6 months
• • 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.
• • Pregnant or breastfeeding patients
• • 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)
• • Patients with suitable 8/8 HLA matched related and unrelated donors
• • 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