Better outcomes of modified myeloablative conditioning without antithymocyte globulin versus myeloablative conditioning in cord blood transplantation for hematological malignancies: A retrospective (development) and a prospective (validation) study

Int J Cancer. 2018 Aug 1;143(3):699-708. doi: 10.1002/ijc.31339. Epub 2018 Mar 12.

Abstract

Cord blood transplantation (CBT) is an effective option for treating hematological malignancies, but graft failure (GF) remains the primary cause of therapy failure. Thus, based on myeloablative conditioning (MAC) of busulfan with cyclophosphamide (Bu/Cy) or total body irradiation with Cy (TBI/Cy), fludarabine (Flu) was added to Bu/Cy and cytarabine (CA) to TBI/Cy for a modified myeloablative conditioning (MMAC). To compare the prognosis of MMAC with MAC, we conducted a retrospective study including 58 patients who underwent CBT with MAC or MMAC from 2000 to 2011. Neutrophil and platelet engraftment rate, overall survival (OS) and disease free survival (DFS) were significantly higher in the MMAC group (adjusted hazard ratio [HR], 2.58, 2.43, 0.36 and 0.37; p < 0.01, p = 0.01, p = 0.02 and p = 0.02, separately). Nonrelapse mortality (NRM) was comparable (p = 0.183). To validate the outcomes noted in the MMAC group, we conducted a prospective single-arm clinical trial including 188 patients who underwent CBT with MMAC from 2011 to 2015. Engraftment rate, survival and NRM of the MMAC group in the prospective trail (MMAC-P) were similar to the MMAC group in the retrospective study (MMAC-R). This study is the first to demonstrate the superiority of MMAC to MAC in CBT for hematological malignancies.

Keywords: antithymocyte globulin; cord blood transplantation; engraftment; hematological malignancies; myeloablative conditioning.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Antilymphocyte Serum / therapeutic use*
  • Blood Platelets
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation* / adverse effects
  • Cord Blood Stem Cell Transplantation* / methods
  • Female
  • Graft vs Host Disease / etiology
  • Hematologic Neoplasms / complications
  • Hematologic Neoplasms / mortality*
  • Hematologic Neoplasms / therapy*
  • Humans
  • Male
  • Middle Aged
  • Neutrophils
  • Retrospective Studies
  • Transplantation Conditioning* / adverse effects
  • Transplantation Conditioning* / methods
  • Treatment Outcome
  • Young Adult

Substances

  • Antilymphocyte Serum