TY - JOUR
T1 - Multilineage hematopoietic engraftment after allogeneic peripheral blood stem cell transplantation without conditioning in SCID patients
AU - Bielorai, Bella
AU - Trakhtenbrot, L.
AU - Amariglio, N.
AU - Rothman, R.
AU - Tabori, U.
AU - Dallal, I.
AU - Golan, H.
AU - Neumann, Y.
AU - Reichart, M.
AU - Kaplinsky, C.
AU - Rechavi, G.
AU - Toren, A.
PY - 2004/8
Y1 - 2004/8
N2 - 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.
AB - 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.
KW - Chimerism
KW - Engraftment
KW - Hematopoietic stem cell transplantation
KW - Severe combined immunodeficiency (SCID)
UR - http://www.scopus.com/inward/record.url?scp=4444283396&partnerID=8YFLogxK
U2 - 10.1038/sj.bmt.1704565
DO - 10.1038/sj.bmt.1704565
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C2 - 15220954
AN - SCOPUS:4444283396
SN - 0268-3369
VL - 34
SP - 317
EP - 320
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
IS - 4
ER -