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BMT12108, Non-Myeloablative Conditioning and Bone Marrow Transplantation

  • Status
    Accepting Candidates
  • Age
    1 Year - 70 Years
  • Sexes
  • Healthy Volunteers
    Accepts Healthy Volunteers


Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.


Full study title A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Disease and Other Hemoglobinopathies
Protocol number OCR20920 ID NCT01850108
Phase N/A



  • Patients who are ineligible for BMT from an HLA-matched sibling donor can proceed to a haplo-BMT. Patients with an HLA-matched related donor will proceed to a matched BMT.

  • Age 1-70 years

  • Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)

  • Patients and donors must be able to sign consent forms. First degree relative 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 anemia such as sickle cell anemia (Hb SS), Hb Sβ° thalassemia, Hb Sβ+thalassemia, Hb SC disease, Hb SE disease, Hb SD disease, Hemoglobin SO* Arab disease HbS with hereditary persistence of fetal hemoglobin. Other significant hemoglobinopathies.

Plus one of the following:

  • Attenuation of progressive disease (adults):

  • Severe and debilitating vaso-occlusive pain despite hydroxyurea or regular blood transfusion therapy.

  • Stroke and silent infarct; stroke or central nervous system event lasting more than 24 hours; MRI changes indicative of brain parenchyma damage and MRA evidence of cerebrovascular disease.

  • Recurrent acute chest syndrome requiring exchange hospitalization.

  • Chronic lung disease as defined by progressive restrictive disease irrespective of oxygen requirements.

  • Chronic kidney disease, CKD stage II * IV

  • Transfusion dépendent thalassemia

Recipient Exclusion Criteria:

  • Poor performance status (ECOG>1).

  • Poor cardiac function: left ventricular ejection fraction

Lead researchers

  • Pediatric Hematologist/Oncologist (Child Cancer Specialist)
    Biljana Horn
  • Pediatric Hematologist/Oncologist (Child Cancer Specialist)

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  1. Step

    Contact the research team

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    Primary contact

  2. Step

    Get screened to confirm eligibility

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  3. Step

    Provide your consent to participate

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  4. Step


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