Multilineage hematopoietic engraftment after allogeneic peripheral blood stem cell transplantation without conditioning in SCID patients

Bella Bielorai, L. Trakhtenbrot, N. Amariglio, R. Rothman, U. Tabori, I. Dallal, H. Golan, Y. Neumann, M. Reichart, C. Kaplinsky, G. Rechavi, A. Toren

Research output: Contribution to journalArticlepeer-review

12 Scopus citations

Abstract

Successful stem cell transplantation for patients with severe combined immunodeficiency (SCID) from matched family donors without conditioning results in engraftment of T lymphocytes. B lymphocytes engraft in only 50% of the cases, while myelopoiesis and erythropoiesis remain of host origin. Full hematopoietic engraftment was reported in one case after bone marrow transplantation without conditioning for a SCID patient. We studied three SCID patients who were transplanted with unmodified mobilized peripheral blood from HLA-identical family sex-mismatched members. They received megadoses of stem cells (18-23 × 106 CD34/kg). In contrast to the expected mixed chimerism that usually occurs in the absence of conditioning, we found in our patients 100% donor cell engraftment based on fluorescence in situ hybridization (FISH) and microsatellite techniques. Subset analysis of the engrafted cells using a multiparametric system enabling a combined analysis of morphology, immunophenotyping and FISH showed that both T and B lymphocytes and myeloid cells were of donor origin in two patients, while T lymphocytes and myeloid cells were of donor origin in the third. In the two cases with ABO incompatibility, erythroid engraftment was evidenced by blood group conversion from recipient to donor type. Multilineage donor engraftment is possible in SCID patients even without conditioning.

Original languageEnglish
Pages (from-to)317-320
Number of pages4
JournalBone Marrow Transplantation
Volume34
Issue number4
DOIs
StatePublished - Aug 2004
Externally publishedYes

Keywords

  • Chimerism
  • Engraftment
  • Hematopoietic stem cell transplantation
  • Severe combined immunodeficiency (SCID)

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