What is the role of HSCT in the treatment of pediatric acute lymphoblastic leukemia (ALL)?

Updated: Jan 02, 2019
  • Author: Vikramjit S Kanwar, MBBS, MBA, MRCP(UK), FAAP; Chief Editor: Jennifer Reikes Willert, MD  more...
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HSCT has been used in very high risk patients in first remission (CR1) as well as in patients with ALL relapse at high risk for further relapse (eg, early BM relapse). Although most patients with relapse achieve second remission (CR2), because two thirds of patients with early relapse eventually have a second relapse, this makes HSCT a recommended option for this group of patients. The improved outcomes of VHR ALL for some categories of patients, such as Ph+ ALL receiving chemotherapy incorporating imatinib, means the role of HSCT in patients with VHR ALL is still debated. [19]

In a collaborative study between the COG and the Center for International Blood and Marrow Transplant Research (CIBMTR), Eapen et al studied 374 children with ALL in CR2 after a marrow relapse who received either a matched sibling donor hematopoietic stem cell transplant (MSD HSCT) (n=186) or ongoing chemotherapy (n=188). [30] The study confirmed better leukemia-free survival in patients with early relapse who received total body irradiation (TBI) based conditioning regimens. The presence of MRD before HSCT is a negative predictor of outcome after HSCT; however, whether aggressive attempts to reduce MRD before HSCT translate into improved long-term survival remains unclear.

Similarly, in the ALL-REZ BFM 90 trial, MSD HSCT benefited patients with higher risk relapse (10-year EFS 40% vs 20% for chemotherapy alone) but did not improve 10-year EFS for lower-risk patients (10-year EFS 52% vs 49% for chemotherapy alone).

With advances in HSCT technique and supportive care, alternative donors (eg, matched unrelated donors) can also be used with equivalent survival outcomes if a MSD is not available.

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