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A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Disease and Other Hemoglobinopathies

Phase 2
2 Years
70 Years
Not Enrolling
Sickle Cell Disease, Hemoglobinopathies

Thank you

Trial Information

A Non-Myeloablative Conditioning and Transplantation of Partially HLA-Mismatched and HLA-Matched Bone Marrow for Patients With Sickle Cell Disease and Other Hemoglobinopathies

Inclusion Criteria:

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 2-70 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 that also fulfill criterion from

Plus one of the following:

1. Stroke or central nervous system event lasting more than 24 hours.

2. MRI changes indicative of brain parenchyma damage.

3. MRA evidence of cerebrovascular disease.

4. Acute chest syndrome requiring exchange transfusion or hospitalization.

5. Recurrent vaso-occlusive pain episodes and hospitalization crisis (more than 2/year
for the last 2 years).

6. Stage I or II sickle lung disease.

7. Transfusion dependent thalassemia

Exclusion Criteria:

Poor performance status (ECOG>1).

- Poor cardiac function: left ventricular ejection fraction<35%.

- Poor pulmonary function: FEV1 and FVC<40% predicted.

- Pulmonary hypertension moderate to severe by echocardiographic standards.

- Poor liver function: direct bilirubin >3.1 mg/dl

- HIV-positive

- Minor (donor anti-recipient) ABO incompatibility if an ABO compatible donor is

- Prior transfusions from donor or recipient if caused alloimmunization vs. donor

- 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.

Criteria for donor eligibility

- Weight ≥ 20kg and age ≥ 18 years

- Donors must meet the selection criteria as defined by the Foundation for the
Accreditation of Hematopoietic Cell Therapy (FAHCT) and will be screened per the
American Association of Blood Banks (AABB). (AABB guidelines and the recipients will
be informed of any deviations.)

- When more than one donor is available, the donor with the lowest number of HLA allele
mismatches will be chosen, unless there is HLA cross-match incompatibility or a
medical reason to select otherwise, in which case donor selection is the
responsibility of the PI, in consultation with the immunogenetics laboratory. In
cases where there is more than one donor with the least degree of mismatch,donors
will be selected based on the most favorable combination of (i) HLA compatibility in
cross-match testing and (ii) ABO compatibility:

- HLA crossmatching (in order of priority)

1. Mutually compatible (no cross-matching antibodies)

2. Recipient non-cross-reactive with donor, donor cross-reactive with recipient

3. Mutually cross-reactive

- ABO compatibility (in order of priority)

- Compatible

- Major incompatibility

- Minor incompatibility

- Major and minor incompatibility

- Donors will be selected to minimize HLA mismatch in the host-versus-graft direction.

- Donor must have a hemoglobin S =/< ~50%.

Type of Study:


Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Transplant-related mortality (TRM)

Outcome Description:

Defined as death in the absence of recurrent sickle cell disease or hemoglobinopathy

Outcome Time Frame:

at 1 year after BMT

Safety Issue:


Principal Investigator

Adetola A Kassim, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Vanderbilt-Ingram Cancer Center


United States: Food and Drug Administration

Study ID:




Start Date:

May 2013

Completion Date:

May 2016

Related Keywords:

  • Sickle Cell Disease
  • Hemoglobinopathies
  • Anemia, Sickle Cell
  • Hemoglobinopathies



Vanderbilt-Ingram Cancer CenterNashville, Tennessee  37232-6838