Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Launched by NORTHSIDE HOSPITAL, INC. · Jul 6, 2016

Keywords

ClinConnect Summary

This clinical trial is studying a new approach to treat severe aplastic anemia, a serious condition where the bone marrow doesn't produce enough blood cells, increasing the risk of infections and bleeding. The researchers are looking at using a type of bone marrow transplant from a partially matched donor (called a haploidentical donor) combined with a medication called cyclophosphamide after the transplant. This method aims to improve the chances of recovery for patients who don't have a perfectly matched sibling donor, which can offer higher survival rates.

To be eligible for this trial, participants should be under 65 years old if they have been treated before, or under 75 years old if they have not. They also need to have a family member who is a partially matched donor and show specific blood and bone marrow conditions. Participants can expect close monitoring throughout the process and access to a potentially effective treatment option. It's important to note that individuals with certain serious health issues, like severe heart or lung problems, may not qualify for the study.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
• • Age \<= 65 years for previously treated and \<= 75 years for previously treated patients
• • KPS \>= 70%
• * Aplastic Anemia that meets the following criteria:
• Peripheral Blood (must fulfill 2 of 3):
• • \<500 PMN/mm3
• • \<20,000 platelets
• • absolute reticulocyte count \<40,000/microL
• Bone Marrow (must be either):
• • markedly hypocellular (\<25% of normal cellularity)
• • moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
• Exclusion Criteria:
• • poor cardiac function (LVEF \<40%)
• • poor pulmonary function (FEV1 \& FVC \<50% predicted)
• • poor liver function (bili \>= 2mg/dL)
• • poor renal function (creatinine \>= 2.0mg/dL or creatinine clearance \<40mL/min)
• • prior allogeneic transplant