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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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Second haploidentical stem cell transplantation for primary graft failure.

Sabrina Giammarco1, Anna Maria Raiola2, Carmen Di Grazia2

  • 1Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma, Italy. sabrina_giammarco@libero.it.

Bone Marrow Transplantation
|December 17, 2020
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Summary

Patients experiencing primary graft failure after a haploidentical bone marrow transplant can be successfully rescued with a second early haploidentical transplant. This approach, using either the same or a different donor, offers a viable solution for graft failure in haploidentical transplants.

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Area of Science:

  • Hematology
  • Transplantation Immunology
  • Oncology

Background:

  • Primary graft failure (PrGF) is a significant complication following haploidentical (HAPLO) unmanipulated bone marrow transplantation.
  • Salvage strategies for PrGF are crucial for improving patient outcomes in HAPLO HSCT.

Purpose of the Study:

  • To evaluate the efficacy of a second HAPLO transplant in patients who experienced PrGF after initial HAPLO HSCT.
  • To identify factors influencing engraftment and survival after a second HAPLO transplant for PrGF.

Main Methods:

  • Retrospective analysis of 19 patients who underwent a second HAPLO transplant for PrGF.
  • Conditioning regimens included total body irradiation (TBI) or fludarabine, busulfan, and thiotepa (TBF).
  • Graft-versus-host disease (GvHD) prophylaxis involved post-transplant cyclophosphamide (PTCY), cyclosporine, and mycophenolate.

Main Results:

  • The second HAPLO transplant achieved trilineage recovery in 74% (14/19) of patients.
  • One-year survival rate was 66% (13/19) after the second HAPLO transplant.
  • Engraftment was observed in 7/8 patients with and 5/7 patients without donor-specific antibodies (DSA).

Conclusions:

  • A second early HAPLO transplant is an effective rescue strategy for patients with PrGF after initial HAPLO HSCT.
  • The use of the same or a different HAPLO donor is feasible for salvage transplantation.
  • Further research may explore optimal conditioning and GvHD prophylaxis for second HAPLO transplants.