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Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan
by
Raheel, Iftikhar
, Hussain Fayyaz
, Chaudhry Qamar un Nisa
, Rafae Abdul
, Shamshad, Ghassan Umair
, Sundas, Ali
, Farhan, Muhammad
, Anwer Faiz
, Ghafoor Tariq
, Parvez, Ahmed
, Rehman Jahanzeb
, Khattak, Tariq Azam
, Khan, Maryam
, Mahmood Syed Kamran
, Shah Zunaira
, Khan, Mehreen Ali
, Shahbaz Nighat
in
Bleeding
/ Bone marrow
/ Conditioning
/ Failure analysis
/ Graft rejection
/ Graft-versus-host reaction
/ Grafts
/ Hematological diseases
/ Hematology
/ Hematopoietic stem cells
/ Metabolic disorders
/ Metabolism
/ Severe combined immunodeficiency
/ Stem cell transplantation
/ Stem cells
/ Survival
2021
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Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan
by
Raheel, Iftikhar
, Hussain Fayyaz
, Chaudhry Qamar un Nisa
, Rafae Abdul
, Shamshad, Ghassan Umair
, Sundas, Ali
, Farhan, Muhammad
, Anwer Faiz
, Ghafoor Tariq
, Parvez, Ahmed
, Rehman Jahanzeb
, Khattak, Tariq Azam
, Khan, Maryam
, Mahmood Syed Kamran
, Shah Zunaira
, Khan, Mehreen Ali
, Shahbaz Nighat
in
Bleeding
/ Bone marrow
/ Conditioning
/ Failure analysis
/ Graft rejection
/ Graft-versus-host reaction
/ Grafts
/ Hematological diseases
/ Hematology
/ Hematopoietic stem cells
/ Metabolic disorders
/ Metabolism
/ Severe combined immunodeficiency
/ Stem cell transplantation
/ Stem cells
/ Survival
2021
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Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan
by
Raheel, Iftikhar
, Hussain Fayyaz
, Chaudhry Qamar un Nisa
, Rafae Abdul
, Shamshad, Ghassan Umair
, Sundas, Ali
, Farhan, Muhammad
, Anwer Faiz
, Ghafoor Tariq
, Parvez, Ahmed
, Rehman Jahanzeb
, Khattak, Tariq Azam
, Khan, Maryam
, Mahmood Syed Kamran
, Shah Zunaira
, Khan, Mehreen Ali
, Shahbaz Nighat
in
Bleeding
/ Bone marrow
/ Conditioning
/ Failure analysis
/ Graft rejection
/ Graft-versus-host reaction
/ Grafts
/ Hematological diseases
/ Hematology
/ Hematopoietic stem cells
/ Metabolic disorders
/ Metabolism
/ Severe combined immunodeficiency
/ Stem cell transplantation
/ Stem cells
/ Survival
2021
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Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan
Journal Article
Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan
2021
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Overview
Management of rare hematological disorders pose unique diagnostic and therapeutic challenges due to unusual occurrence and limited treatment options. We retrospectively identified 45 patients receiving matched related donor transplant for rare hematological disorders from 2006 to 2019. Patients were divided into two groups (1) malignant and (2) non malignant. The malignant disorder group included four patients while the nonmalignant group included 41 patients divided into immune dysregulation (n = 23), bone marrow failure (n = 10), metabolic (n = 5), and bleeding diathesis (n = 3). Twenty-six (57.8%) patients received myeloablative conditioning (MAC) and 16 (35.6%) received reduced intensity conditioning (RIC), while 3 (6.6%) patients with severe combined immunodeficiency received stem cell infusion alone without conditioning. The cumulative incidence (CI) of grade II–IV acute GVHD (aGVHD) was 39.1% (n = 18) and chronic GVHD (cGVHD) 15.2% (n = 7). There was no primary graft failure while CI of secondary graft failure was 9%. Overall survival (OS) and disease-free survival (DFS) was 82.2% and 77.8% respectively. Group wise OS was 75% in the malignant group, 82.6% in the immune dysregulation group, 80% in patients with metabolic disorders and bone marrow failure, while 100% in patients with bleeding diathesis. This retrospective analysis shows that hematopoietic stem cell transplant can be a feasible treatment option for rare hematological disorders.
Publisher
Nature Publishing Group
Subject
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