Severe Aplastic Anemia Clinical Trial
Official title:
Outcome of Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin (ATG ) Conditioning Regimens in Severe Aplastic Anemia Patients
Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia.The theoretical basis for marrow failure includes primary defects in or damage to the stem cell or the marrow microenvironment
The distinction between acquired and inherited disease may present a clinical challenge, but
more than 80% of cases are acquired.
Therapy for aplastic anemia may consist of supportive care only, immunosuppressive therapy,
or hematopoietic cell transplantation. Severe and very severe aplastic anemia have a high
mortality rate with supportive care alone .
The British Committee for Standards in Haematology recommends treating infection or
uncontrolled bleeding before administering immunosuppressive therapy, including in patients
scheduled for hematopoietic cell transplantation. The Pediatric Haemato-Oncology Italian
Association recommends hematopoietic cell transplantation from a matched sibling donor for
severe aplastic anemia, and if a matched donor is not available, options include
immunosuppressive therapy or unrelated donor hematopoietic cell transplantation.
Human leukocyte antigen (HLA)-matched sibling-donor hematopoietic cell transplantation is the
treatment of choice for a young patient with severe or very severe aplastic anemia , being
generally accepted for patients younger than 40 years.
Recent years have seen increasing the use of hematopoietic cells other than bone marrow (BM).
These alternative graft sources include peripheral blood progenitor cells and granulocyte
colony stimulating factor (G-CSF) bone marrow (G-BM). Several groups have demonstrated that
peripheral blood progenitor cell transplantation has faster neutrophil and platelet
engraftment compared to BM in patients with hematologic malignancies ; however, most adult
studies also report an increase in chronic GVHD (cGVHD) Some adult studies describe improved
survival with peripheral blood progenitor cells in adult recipients although survival
generally was no different in those with standard-risk disease .
Cyclophosphamide (Cy)/anti-thymocyte globulin (ATG) is considered the standard conditioning
regimen for patients with severe aplastic anemia undergoing hematopoietic cell
transplantation from a HLA matched sibling donor, The introduction of a fludarabine (F-araA)
based reduced intensity conditioning regimen has extended the availability of hematopoietic
cell transplantation to patients who are older, heavily transfused and having delayed
treatment from the time of diagnosis with HLA matched related/unrelated donors.
The addition of F-araA to the conditioning regimen has been shown to provide additional
immunosuppression for engraftment without increasing toxicity in patients undergoing
hematopoietic cell transplantation .
Also, conditioning with F-araA and Cy is associated with improved long-term survival compared
to a historical cohort receiving Cy/ATG regimen in patients with severe aplastic anemia
undergoing hematopoietic cell transplantation .
Adequate post transplantation immunosuppression is important not only for the prevention of
GVHD, but also to secure adequate suppression of the host immune system and prevention of
graft rejection. The administration of CsA alone or with or without short-course methotrexate
or steroid should be considered the standard post transplantation immunosuppression. In
addition to possibility of use of other immunosuppressive agents, including the use of
mycophenolate mofetil, particularly in patients with renal impairment.
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