Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies

Raheel Iftikhar, Qamar un Nisa Chaudhry, Faiz Anwer, Karun Neupane, Abdul Rafae, Syed Kamran Mahmood, Tariq Ghafoor, Nighat Shahbaz, Mehreen Ali Khan, Tariq Azam Khattak, Ghassan Umair Shamshad, Jahanzeb Rehman, Muhammad Farhan, Maryam Khan, Iqraa Ansar, Rabia Ashraf, Judith Marsh, Tariq Mehmood Satti, Parvez Ahmed

Research output: Contribution to journalReview articlepeer-review

4 Scopus citations


Treatment options for newly diagnosed aplastic anemia (AA) patient includes upfront allogeneic hematopoietic stem cell transplant (HSCT) or immunosuppressive therapy (IST). With recent advances in supportive care, conditioning regimens and post-transplant immunosuppression the overall survival for HSCT approaches 70–90%. Transplant eligibility needs to be assessed considering age, comorbidities, donor availability and probability of response to immunosuppressive therapy (IST). Upfront HSCT should be offered to children and young adults with matched related donor (MRD). Upfront HSCT may also be offered to children and young adults with rapidly available matched unrelated donor (MUD) who require urgent HSCT. Bone marrow (BM) graft source and cyclosporine (CsA) plus methotrexate (MTX) as graft versus host disease (GVHD) prophylaxis are preferable when using anti-thymocyte globulin (ATG) based conditioning regimens. Alemtuzumab is an acceptable alternative to ATG and is used with CsA alone and with either BM or peripheral blood stem cells (PBSC). Cyclophosphamide (CY) plus ATG conditioning is preferable for patients receiving MRD transplant, while Fludarabine (Flu) based conditioning is reserved for older adults, those with risk factors of graft failure and those receiving MUD HSCT. For haploidentical transplant, use of low dose radiotherapy and post-transplant cyclophosphamide has resulted in a marked reduction in graft failure and GVHD.

Original languageEnglish (US)
Article number100772
JournalBlood Reviews
StatePublished - May 2021
Externally publishedYes


  • Aplastic anemia
  • Graft vs host disease
  • Peripheral blood stem cells
  • Stem cell transplantation

ASJC Scopus subject areas

  • Hematology
  • Oncology


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