HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy

Launched by CHILDREN'S HOSPITAL MEDICAL CENTER, CINCINNATI · May 16, 2014

Keywords

ClinConnect Summary

This clinical trial is studying a new approach to help patients with Fanconi Anemia (FA) by using a type of treatment called stem cell transplant. The goal is to see if using lower doses of a chemotherapy drug called busulfan, while not using another medication called cyclosporine, can lead to fewer side effects after the transplant. Patients will receive cells from either a related donor who is not a fully matched sibling or an unrelated donor. This study aims to help those with severe blood problems linked to FA, such as severe marrow failure, myelodysplastic syndrome (MDS), or acute myelogenous leukemia (AML).

To be eligible for this trial, patients need to have a confirmed diagnosis of Fanconi Anemia and meet certain health criteria regarding their blood counts and overall physical condition. Participants will undergo a preparative treatment before the transplant and will need to agree to be part of this research by signing a consent form. It's also important for female patients and donors to not be pregnant or breastfeeding when joining the study. This trial is currently looking for participants, and it offers a chance to receive a specialized treatment that could improve health outcomes for people facing these serious conditions.

Gender

ALL

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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