Latin American consensus on the supportive management of patients with severe combined immunodeficiency
Date
2019
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Article
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Abstract
Severe combined immunodeficiency (SCID) represents the most lethal form of primary immunodeficiency, with mortality rates of greater than 90% within the first year of life without treatment. Hematopoietic stem cell transplantation and gene therapy are the only curative treatments available, and the best-known prognostic factors for success are age at diagnosis, age at hematopoietic stem cell transplantation, and the comorbidities that develop in between. There are no evidence-based guidelines for standardized clinical care for patients with SCID during the time between diagnosis and definitive treatment, and we aim to generate a consensus management strategy on the supportive care of patients with SCID. First, we gathered available information about SCID diagnostic and therapeutic guidelines, then we developed a document including diagnostic and therapeutic interventions, and finally we submitted the interventions for expert consensus through a modified Delphi technique. Interventions are grouped in 10 topic domains, including 123 “agreed” and 38 “nonagreed” statements. This document intends to standardize supportive clinical care of patients with SCID from diagnosis to definitive treatment, reduce disease burden, and ultimately improve prognosis, particularly in countries where newborn screening for SCID is not universally available and delayed diagnosis is the rule. Our work intends to provide a tool not only for immunologists but also for primary care physicians and other specialists involved in the care of patients with SCID.
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Citation
Juan Carlos Bustamante Ogando, Armando Partida Gaytán, Juan Carlos Aldave Becerra, Aristóteles Álvarez Cardona, Liliana Bezrodnik, Arturo Borzutzky, Lizbeth Blancas Galicia, Diana Cabanillas, Antonio Condino-Neto, Agustín De Colsa Ranero, Sara Espinosa Padilla, Juliana Folloni Fernandes, Jorge Alberto García Campos, Héctor Gómez Tello, María Edith González Serrano, Alonso Gutiérrez Hernández, Víctor Manuel Hernández Bautista, Gabriele Ivankovich Escoto, Alejandra King, Juliana Lessa Mazzucchelli, Beatriz Adriana Llamas Guillén, Saul Oswaldo Lugo Reyes, Sarbelio Moreno Espinosa, Matías Oleastro, Francisco Otero Mendoza, María Cecilia Poli Harlowe, Oscar Porras, Nideshda Ramirez Uribe, Lorean Regairaz, Francisco Rivas Larrauri, Federico José Saracho Weber, Anete S. Grumach, Tamara Staines Boone, Beatriz Tavares Costa-Carvalho, Marco Antonio Yamazaki Nakashimada, Francisco Javier Espinosa Rosales, Latin American consensus on the supportive management of patients with severe combined immunodeficiency, Journal of Allergy and Clinical Immunology, Volume 144, Issue 4, 2019, Pages 897-905, ISSN 0091-6749, https://doi.org/10.1016/j.jaci.2019.08.002.
Keywords
Severe combined immunodeficiency, Consensus, Trans- plantation, Supportive measures, Treatment, Immunodeficiency, Transplantation, primary immunodeficiency