HLA-haploidentical umbilical cord blood stem cell transplantation in a child with advanced leukemia : clinical outcome and analysis of hematopoietic recovery

• Published in: Bone marrow transplantation (Basingstoke) Vol. 16; no. 2; pp. 229 - 240
• Main Authors: MINIERO, R, BUSCA, A, SARACCO, P, RUGGIERI, L, VASSALLO, E, GABUTTI, V, MADON, E, RONCAROLO, M. G, SAITTA, M, IAVARONE, A, TIMEUS, F, BIONDI, A, AMOROSO, A, PERUGINI, L, CIUTI, E
• Format: Journal Article
• Language: English
• Published: Basingstoke Nature Publishing Group 01.08.1995
• Subjects: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy; Biological and medical sciences; Bone marrow, stem cells transplantation. Graft versus host reaction; Child, Preschool; Chimera; Fetal Blood - cytology; Haploidy; Hematopoietic Stem Cell Transplantation; Histocompatibility Testing; Humans; Male; Medical sciences; Precursor Cell Lymphoblastic Leukemia-Lymphoma - therapy; Transfusions. Complications. Transfusion reactions. Cell and gene therapy; Human; Transfusion; Prognosis; Acute; Stem cell; Related donor; Hematopoietic cell; Homograft; Malignant hemopathy; Case study; Treatment; Lymphoproliferative syndrome; Advanced stage; Graft; Umbilical cord; Acute lymphocytic leukemia; Child; Haplotype; Histocompatibility
• ISSN: 0268-3369; 1476-5365

Growing attention has been focused on cord blood as a source of transplantable hematopoietic stem cells. However, clinical experience is rather limited. In this study we describe a child with advanced acute lymphoblastic leukemia who received an HLA-haploidentical cord blood transplant. The patient was transplanted in third complete remission after conditioning with fractionated total body irradiation, thiotepa and cyclophosphamide. Forty-one milliliters of cryopreserved umbilical cord blood, containing 0.15 x 10(8) nucleated cells/kg and 0.25 x 10(4) CFU-GM/kg, were infused. Cyclosporine and prednisone were administered for graft-versus-host disease (GVHD) prophylaxis. The patient received G-CSF from day +1 to day +35, but no improvement in granulocyte counts was observed. Therefore, administration of GM-CSF was started on day +36 to day +59, which resulted in a significant increase in white blood cells and granulocyte counts. Sustained myeloid engraftment was evidenced by a granulocyte count > 0.5 x 10(9)/l by day +41. The presence of donor-derived cells could be documented in the peripheral blood and bone marrow of the patient by cytogenetic analysis, HLA phenotyping and DNA studies. Forty-one days after transplant, clonogenic bone marrow assays showed the presence of low frequencies of primitive hematopoietic progenitor cells (BFU-E = 19/10(5) and CFU-GM = 8/10(5)). The chimerism was complete and no host-derived cells could be detected. However, the engraftment was restricted to the myeloid lineage whereas lymphoid and megakaryocytic engraftments were inadequate. The immunophenotype of the patient's peripheral blood showed the presence of T lymphocytes expressing an immature phenotype (CD2+ CD3-) at day +21.