HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Description

The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.

Conditions

Fanconi Anemia, Severe Marrow Failure, Myelodysplastic Syndrome (MDS), Acute Myelogenous Leukemia (AML)

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Study Overview

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Study Details

Study overview

The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.

A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Condition
Fanconi Anemia
Intervention / Treatment

-

Contacts and Locations

New York

Memorial Sloan Kettering Cancer Center, New York, New York, United States, 10174

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Seattle

Fred Hutchinson Cancer Research Center, Seattle, Washington, United States, 98109

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Patients must have a diagnosis of Fanconi anemia
  • * Patients must have one of the following hematologic diagnoses:
  • 1. Severe Aplastic Anemia (SAA), with bone marrow cellularity of \<25% OR Severe Isolated Single Lineage Cytopenia and at least one of the following features:
  • 1. Platelet count \<20 x 109/L or platelet transfusion dependence\*
  • 2. ANC \<1000 x 109/L
  • 3. Hgb \<8 gm/dl or red cell transfusion dependence\*
  • 2. Myelodysplastic Syndrome (MDS) (based on WHO or IPSS Classification
  • 3. Acute Myelogenous Leukemia (untreated, in remission or with refractory or relapsed disease)
  • * Donors will be either human leukocyte antigen (HLA) compatible unrelated or HLA-genotypically matched related donors (no fully matched sibling donor).
  • * Patients and donors may be of either gender or any ethnic background.
  • * Patients must have a Karnofsky adult, or Lansky pediatric performance scale status \> 70%.
  • * Patients must have adequate physical function measured by:
  • 1. Cardiac: asymptomatic or if symptomatic then 1) left ventricular ejection fraction (LVEF) at rest must be \> 50% and must improve with exercise or 2) Shortening Fraction \> 29%
  • 2. Hepatic: \< 5 x upper limit of normal (ULN) alanine transaminase (ALT) and \< 2.0 mg/dl total serum bilirubin.
  • 3. Renal: serum creatinine \<1.5 mg/dl or if serum creatinine is outside the normal range, then CrCl \> 50 ml/min/1.73 m2
  • 4. Pulmonary: asymptomatic or if symptomatic, DLCO \> 50% of predicted
  • * Each patient must be willing to participate as a research subject and must sign an informed consent form.
  • * Female patients and donors must not be pregnant or breastfeeding at the time of signing consent. Women must be willing to undergo a pregnancy test prior to transplant and avoid becoming pregnant while on study.
  • * Active CNS leukemia
  • * Female patients who are pregnant (positive serum or urine HCG) or breast-feeding.
  • * Active uncontrolled viral, bacterial or fungal infection
  • * Patient seropositive for HIV-I/II; HTLV -I/II

Ages Eligible for Study

3 Months to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Children's Hospital Medical Center, Cincinnati,

Parinda Mehta, MD, PRINCIPAL_INVESTIGATOR, CCHMC

Study Record Dates

2027-07