BACKGROUND: Currently, hematopoietic stem cell transplantation allows long-term survival in a high proportion of infants with congenital severe T-cell immunodeficiency. However, relatively little is known of their long-term quality of life. OBJECTIVE: We sought to assess the long-term immune reconstitution and clinical status in children treated with stem cell transplantation for severe T-cell immunodeficiency. METHODS: Immune function and clinical status have been analyzed in a cohort of 40 patients with severe T-cell immunodeficiency who are alive at a follow-up of at least 5 years after transplantation. RESULTS: Most patients have attained normal T- and B-cell function. Weight and height were normal at last follow-up in most patients. Endocrine and severe neurologic abnormalities have been observed in 17.5% and 10% of the patients, respectively. CONCLUSIONS: These data indicate that with current management strategies, stem cell transplantation can lead to long-term survival and good quality of life in the majority of patients with severe T-cell immunodeficiency. CLINICAL IMPLICATIONS: Prompt recognition of congenital severe T-cell immunodeficiency, followed by stem cell transplantation, allows excellent perspectives of long-term survival and good quality of life for these otherwise fatal disorders.
BACKGROUND: Currently, hematopoietic stem cell transplantation allows long-term survival in a high proportion of infants with congenital severe T-cell immunodeficiency. However, relatively little is known of their long-term quality of life. OBJECTIVE: We sought to assess the long-term immune reconstitution and clinical status in children treated with stem cell transplantation for severe T-cell immunodeficiency. METHODS: Immune function and clinical status have been analyzed in a cohort of 40 patients with severe T-cell immunodeficiency who are alive at a follow-up of at least 5 years after transplantation. RESULTS: Most patients have attained normal T- and B-cell function. Weight and height were normal at last follow-up in most patients. Endocrine and severe neurologic abnormalities have been observed in 17.5% and 10% of the patients, respectively. CONCLUSIONS: These data indicate that with current management strategies, stem cell transplantation can lead to long-term survival and good quality of life in the majority of patients with severe T-cell immunodeficiency. CLINICAL IMPLICATIONS: Prompt recognition of congenital severe T-cell immunodeficiency, followed by stem cell transplantation, allows excellent perspectives of long-term survival and good quality of life for these otherwise fatal disorders.
Authors: F Porta; S Cavagnini; L Imberti; A Sottini; F Bolda; A Beghin; A Caruso; A Lanfranchi Journal: Bone Marrow Transplant Date: 2015-09-14 Impact factor: 5.483
Authors: Jennifer Heimall; Jennifer Puck; Rebecca Buckley; Thomas A Fleisher; Andrew R Gennery; Benedicte Neven; Mary Slatter; Elie Haddad; Luigi D Notarangelo; K Scott Baker; Andrew C Dietz; Christine Duncan; Michael A Pulsipher; Mort J Cowan Journal: Biol Blood Marrow Transplant Date: 2017-01-06 Impact factor: 5.742
Authors: Mary Eapen; Kwang Woo Ahn; Paul J Orchard; Morton J Cowan; Stella M Davies; Anders Fasth; Anna Hassebroek; Mouhab Ayas; Carmem Bonfim; Tracey A O'Brien; Thomas G Gross; Mitchell Horwitz; Edwin Horwitz; Neena Kapoor; Joanne Kurtzberg; Navneet Majhail; Olle Ringden; Paul Szabolcs; Paul Veys; K Scott Baker Journal: Biol Blood Marrow Transplant Date: 2012-03-16 Impact factor: 5.742
Authors: Linda M Griffith; Morton J Cowan; Luigi D Notarangelo; Jennifer M Puck; Rebecca H Buckley; Fabio Candotti; Mary Ellen Conley; Thomas A Fleisher; H Bobby Gaspar; Donald B Kohn; Hans D Ochs; Richard J O'Reilly; J Douglas Rizzo; Chaim M Roifman; Trudy N Small; William T Shearer Journal: J Allergy Clin Immunol Date: 2009-12 Impact factor: 10.793
Authors: Linda M Griffith; Morton J Cowan; Donald B Kohn; Luigi D Notarangelo; Jennifer M Puck; Kirk R Schultz; Rebecca H Buckley; Mary Eapen; Naynesh R Kamani; Richard J O'Reilly; Robertson Parkman; Chaim M Roifman; Kathleen E Sullivan; Alexandra H Filipovich; Thomas A Fleisher; William T Shearer Journal: J Allergy Clin Immunol Date: 2008-11-06 Impact factor: 10.793
Authors: William T Shearer; Elizabeth Dunn; Luigi D Notarangelo; Christopher C Dvorak; Jennifer M Puck; Brent R Logan; Linda M Griffith; Donald B Kohn; Richard J O'Reilly; Thomas A Fleisher; Sung-Yun Pai; Caridad A Martinez; Rebecca H Buckley; Morton J Cowan Journal: J Allergy Clin Immunol Date: 2013-11-28 Impact factor: 10.793