Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients

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Brief Title

Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients

Official Title

Outcome of Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin (ATG ) Conditioning Regimens in Severe Aplastic Anemia Patients

Brief Summary

      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
    

Detailed Description

      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.
    

Study Phase

Phase 2/Phase 3

Study Type

Interventional


Primary Outcome

Overall Survival rate

Secondary Outcome

 GVHD occurrence (Acute or Chronic), engraftment and transplant related mortality

Condition

Severe Aplastic Anemia

Intervention

Non ATG Conditioning regimen

Study Arms / Comparison Groups

 Non ATG regimen
Description:  the first 50% of patients will receive Fludarabine + cyclophosphamide prior to hematopoietic cell transplantation. GVHD Prophylaxis: Cyclosporine A All the patient will do allogenic stem cell transplantation from peripheral blood

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

50

Start Date

January 1, 2019

Completion Date

June 2020

Primary Completion Date

December 1, 2019

Eligibility Criteria

        Inclusion Criteria:

          -  All patients with severe and very severe aplastic anemia for stem cell therapy.

        Exclusion Criteria:

        1- Contraindication to stem cell transplantation. 2 - Patients associated co-morbidities.
      

Gender

All

Ages

16 Years - 50 Years

Accepts Healthy Volunteers

No

Contacts

, 00201006372498, [email protected]



Administrative Informations


NCT ID

NCT03295058

Organization ID

OPALLSCT


Responsible Party

Principal Investigator

Study Sponsor

Assiut University


Study Sponsor

, , 


Verification Date

November 2018