Journal Article

B cell function after haploidentical in utero bone marrow transplantation in a patient with severe combined immunodeficiency

Gómez-Pineda, A, Fernández-Cruz, E, Cela, E, Rodríguez-Molina, JJ, Ugazio, AG, Bartolomé, J, Porta, F, Lafranchi, A, Gil, J, Cantalejo, A, Notarangelo, LD

2002

Overview

An in utero paternal CD34(+) cell transplant was performed in a T-B+NK+ SCID fetus. We report here the results of the 3-year humoral immune reconstitution study. The methods used were ApoB VNTR typing, flow cytometry, nephelometry, hemagglutination, ELISA, ELISPOT and lymphoproliferative assays. The T cells were of donor origin whereas monocytes, B and NK cells were of host origin. Peripheral B cell counts and IgM levels were normal since birth. IVIG therapy was required at 5 months of age until 2 years old. IgA levels > or =20 mg/dl were detected from month 17 post transplantation. Isohemagglutinins were present since month 8 post transplantation, the highest titers (anti-A:1/128, anti-B:1/32) were obtained at month 33 post-transplantation. After immunization with rHBsAg, circulating anti-HBsAg IgG secreting cells and a 7.8-fold increase in serum anti-HBsAg Ab were detected. We conclude that split chimerism following in utero haploidentical BMT allows complete humoral immune reconstitution in a T-B+NK+ SCID patient.

• Publisher: Nature Publishing Group
• Subject: Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy / Antibody Formation / Apolipoproteins B - genetics / B-Lymphocytes - cytology / B-Lymphocytes - immunology / Biological and medical sciences / Biomarkers / Bone marrow / Bone marrow transplantation / Bone Marrow Transplantation - methods / Bone marrow, stem cells transplantation. Graft versus host reaction / CD34 antigen / Cell Lineage / Chimerism / Consanguinity / Enzyme-linked immunosorbent assay / Fathers / Fetal Diseases - diagnosis / Fetal Diseases - embryology / Fetal Diseases - genetics / Fetal Diseases - therapy / Fetuses / Flow cytometry / Follow-Up Studies / Graft Survival / Haplotypes - genetics / Hemagglutination / Hepatitis B surface antigen / Histocompatibility / Humans / Immune reconstitution / Immunization / Immunoglobulin A / Immunoglobulin A - biosynthesis / Immunoglobulin G / Immunoglobulin M / Immunophenotyping / Infant, Newborn / Intravenous administration / Living Donors / Lymphocytes / Lymphocytes T / Male / Medical sciences / Minisatellite Repeats / Monocytes / Nephelometry / Prenatal Diagnosis / Severe combined immunodeficiency / Severe Combined Immunodeficiency - diagnosis / Severe Combined Immunodeficiency - embryology / Severe Combined Immunodeficiency - genetics / Severe Combined Immunodeficiency - therapy / Stem cell transplantation / T-Lymphocyte Subsets - cytology / T-Lymphocyte Subsets - immunology / Transfusions. Complications. Transfusion reactions. Cell and gene therapy / Transplantation / Transplantation Chimera - immunology / Transplants & implants / Vaccination