Haploidentical Hematopoietic Stem Cell Transplantation

  • End date
    Apr 20, 2023
  • participants needed
  • sponsor
    Catherine Bollard
Updated on 20 July 2020
Allistair Abraham, MD
Primary Contact
Childrens National Medical Center (5.3 mi away) Contact
cell transplantation
blood transfusion
iron overload
transfusion dependent thalassemia


The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia. The purpose of the study is to assess the safety and toxicity of reduced intensity conditioning haploidentical hematopoietic stem cell transplantation.


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 electromechanical device intended to isolate certain cell subsets from mixed cell populations. When used in combination with the CliniMACs CD34 reagent, it is possible to prepare extremely pure populations of CD34+ cells with upwards of 5 logs depletion of contaminating T cells within a closed and sterile system.

We intend to use this system to select cells from HLA haploidentical related donors who have been mobilized with G-CSF prior to stem cell collection. Since previous investigations of this strategy in adult patients have not translated into enhanced long term survival, we intend to limit this protocol to patients under the age of 22 as they have more rapid immune reconstitution.

Treatment peripheral blood stem cell graft that are CD34+ selected
Clinical Study IdentifierNCT02165007
SponsorCatherine Bollard
Last Modified on20 July 2020

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Inclusion Criteria

Is your age less than or equal to 22 yrs?
Gender: Male or Female
Do you have any of these conditions: Sickle Cell-thalassemia Disease or Thalassemia?
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 FEV150% 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
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