HLA-haploidentical stem cell transplantation after removal of αβ+ T and B cells in children with nonmalignant disorders

Alice Bertaina, Pietro Merli, Sergio Rutella, Daria Pagliara, Maria Ester Bernardo, Riccardo Masetti, Daniela Pende, Michela Falco, Rupert Handgretinger, Francesca Moretta, Barbarella Lucarelli, Letizia P. Brescia, Giuseppina Li Pira, Manuela Testi, Caterina Cancrini, Nabil Kabbara, Rita Carsetti, Andrea Finocchi, Alessandro Moretta, Lorenzo MorettaFranco Locatelli

Research output: Contribution to journalArticle

Abstract

Twenty-three children with nonmalignant disorders received HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT) after ex vivo elimination of αβ+ T cells and CD19+ B cells. The median number of CD34+, αβ+CD3 +, and B cells infusedwas 16.8 × 106, 40 × 103, and 40 × 103 cells/kg, respectively. No patient received any posttransplantation pharmacologic prophylaxis for graft-versus-host disease (GVHD). All but 4 patients engrafted, these latter being rescued by a second allograft. Three patients experienced skin-only grade 1 to 2 acute GVHD. No patient developed visceral acute or chronic GVHD. Cumulative incidence of transplantation-related mortality was 9.3%. With a median follow-up of 18 months, 21 of 23 children are alive and disease-free, the 2-year probability of disease-free survival being 91.1%. Recovery of γδ+ T cells was prompt, but αβ+ T cells progressively ensued over time.Our datasuggest that thisnovelgraftmanipulation strategy is safe and effective for haplo-HSCT. This trial was registered at www.clinicaltrials.gov as #NCT01810120.

Original languageEnglish
Pages (from-to)822-826
Number of pages5
JournalBlood
Volume124
Issue number5
DOIs
Publication statusPublished - Jul 31 2014

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology

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