A Phase 2 interventional study of Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide'' in Aplastic Anemia Idiopathic, sponsored by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan. Status unknown at 1 site in Pakistan. Open to participants aged 2 Years to 60 Years. Per ClinicalTrials.gov, last updated 2020-08-11.
Sponsored by National Institute of Blood and Marrow Transplant (NIBMT), Pakistan · Phase 2, Interventional, and Treatment
Severe and very severe aplastic anemia are life threatening disorders for which allogeneic stem cell transplant is only curative treatment. However, matched sibling donor (MSD) is available in only 25-35% cases. Pakistan has a population of around 203 million but there is no donor registry available so there is no option available for matched unrelated donor (MUD) transplants . Haploidentical transplant represents only curative option for patients lacking MSD. Protocols involving post transplant cyclophosphamide require Total body irradiation (TBI) and utilize peripheral blood stem cell(PBSC) as graft source. TBI is not available in most of transplant centres across Pakistan due to lack of availability , cost and lack of expertise. The investigators have conceived a novel TBI free conditioning regimen to be used for haplo-identical Hemtopoeitic stem cell transplant in acquired aplastic anemia patients
Aplastic anemia is considered to be a rare and heterogenous disease with incidence of 1-2 per million in western countries. Data from Asian studies show a 3-4 fold higher incidence.Majority of newly diagnosed aplastic anemia patients are younger and in Armed forces bone marrow transplant cohort of 1324 patients 64 % patient are younger than 24 years of age and 87% patients younger than 40 years (unpublished data).There is no donor registry in Pakistan and patients lacking sibling match donor cannot proceed to stem cell transplant due lack of matched unrelated donors. Horse antithymocyte globulin is not available currently in Pakistan and response to Rabbit antithymocyte globulin is dismal as shown in number of international studies. So haploidentical stem cell transplant remains only curative option for patients lacking Matched sibling donor. Currently there are 2 major platforms used for haplo-identical stem cell transplant. Post transplant cyclophosphamide based using TBI and haplo regimen of Peking university. TBI is not available for most of our patients in Pakistan due to cost,non-availability and lack of expertise. The investigators have formulated a novel TBI free regimen incorporating Busulphan, antithymocyte globulin and using co-primed bone marrow and peripheral blood harvest to minimize graft-versus-host disease and facilitate engraftment. Post transplant cyclophosphamide, Cyclosporine and mycophenolate mofetil will be used for graft-versus-host disease prophylaxis
1,733 studies on the registry are indexed under Anemia; 246 are open to participants now.
This study's planned enrollment of 30 is below the median of 94 across 1,291 interventional studies indexed under Anemia.
Browse Anemia studies →National Institute of Blood and Marrow Transplant (NIBMT), Pakistan is the lead sponsor of 11 studies on the registry; 3 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Aplastic Anemia that meets the following criteria:
i. Peripheral Blood (must fulfill 2 of 3): ii. \<500 neutrophils iii. \<20,000 platelets iv. absolute reticulocyte count \<40,000/microL
Exclusion Criteria:
Recipients will receive a reduced intensity conditioning regimen of ''Fludarabine'' 30 mg/m2 IV daily from day -7 to -3, ''Cyclophosphamide'' 14.5-30 mg/kg IV daily on day -6 and -5 , ''rabbit Antithymocyte globulin'' 5 mg /kg/day from day -6 to day-3; ''Busulphan'' IV 3.2 mg per kg/day in 02 divided doses on day -3 and day-2, ''Granulocyte Colony Stimulating factor primed Bone marrow harvest'' and/OR ''PBSC'' graft on day 0 and day +1 respectively and Graft versus host disease prophylaxis with ''post-transplant cyclophosphamide'' administered at a dose of 50mg/kg/day given daily on days +3 and +5 post-transplant and ''cyclosporine'' from day +5, ''mycophenolate mofetil'' from day+5 to day+35.
Drug: Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''
''Busulphan'' will be used in place of ''TBI'' in equivalent myelotoxic dose to facilitate engraftment , ''ATG'' will be used to reduce GVHD and facilitate engraftment while ''combine PBSC'' and/OR ''Bone marrow harvest'' will be used
Also known as: ''Combined BMH'' and ''PBSC harvest''
Number of Participants with overall survival
overall survival is defined as the time interval from date of transplant to death or to last follow-up, whichever occurs first.
Time frame: from the date of transplant to 1 year post transplant
Number of Participants with Disease free survival
from time of transplant to death or last follow up
Time frame: from the date of transplant to 1 year post transplant
Time of Neutrophil engraftment
first of 3 consecutive days with Absolute neutrophil count\> 0.5
Time frame: from the date of transplant to 10 to day 28 post transplant
Frequency of Graft versus host disease
as per clinical and histopathological diagnosis
Time frame: from the date of transplant to acute upto 100 day post transplant, chronic >100 days post transplant
Rate of Complications
both infectious and non infectious
Time frame: from the date of transplant to 1 year from day of transplantation
Plan to share: No
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This study is status unknown, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.
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National Institute of Blood and Marrow Transplant (NIBMT), Pakistan