Allogeneic hematopoietic stem cell transplant in rare hematologic disorders: a single center experience from Pakistan

Maryam Khan, Raheel Iftikhar, Tariq Ghafoor, Fayyaz Hussain, Qamar un Nisa Chaudhry, Syed Kamran Mahmood, Nighat Shahbaz, Mehreen Ali Khan, Tariq Azam Khattak, Ghassan Umair Shamshad, Jahanzeb Rehman, Sundas Ali, Zunaira Shah, Abdul Rafae, Muhammad Farhan, Faiz Anwer, Parvez Ahmed

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)863-872
Number of pages10
JournalBone Marrow Transplantation
Issue number4
StatePublished - Apr 2021

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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