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Long-Term Immune Reconstitution in ADA-Deficient Patients Treated With Elapegademase: A Real-World Experience.
Murguia-Favela, Luis; Suresh, Sneha; Wright, Nicola A M; Alvi, Saima; Tehseen, Sarah; Hernandez-Trujillo, Vivian; Seroogy, Christine M; Haddad, Elie; Nieves, Daime; Hershfield, Michael S; Walter, Jolan E; Pettiford, Leah; Kamani, Naynesh R; Keller, Michael D; Pham-Huy, Anne; Grunebaum, Eyal.
Afiliación
  • Murguia-Favela L; Section of Hematology/Immunology, Department of Pediatrics, Alberta Children's Hospital and the University of Calgary, Calgary, Alberta, Canada.
  • Suresh S; Division of IHOPE, Department of Pediatrics, University of Alberta, Stollery Children's Hospital, Edmonton, Alberta, Canada.
  • Wright NAM; Section of Hematology/Immunology, Department of Pediatrics, Alberta Children's Hospital and the University of Calgary, Calgary, Alberta, Canada.
  • Alvi S; Division of Pediatric Hematology/Oncology, Jim Pattison Children's Hospital, Saskatoon, Saskatchewan, Canada.
  • Tehseen S; Division of Hematology/Oncology and Transfusion Medicine, Jim Pattison Children's Hospital, Saskatoon, Saskatchewan, Canada.
  • Hernandez-Trujillo V; Allergy and Immunology Care Center of South Florida, Miami Lakes, Fla.
  • Seroogy CM; Division of Allergy, Immunology & Rheumatology, University of Wisconsin School of Medicine and Public Health, Madison, Wis.
  • Haddad E; Division of Immunology and Rheumatology, Department of Pediatrics, Centre Hospitalier Universitaire (CHU) Sainte-Justine, University of Montreal, Montreal, Quebec, Canada.
  • Nieves D; Division of Pediatric Allergy and Immunology, University of South Florida at Johns Hopkins All Children's Hospital, St Petersburg, Fla.
  • Hershfield MS; Division of Medicine and Biochemistry, Duke University Medical Center, Durham, NC.
  • Walter JE; Division of Pediatric Allergy and Immunology, University of South Florida at Johns Hopkins All Children's Hospital, St Petersburg, Fla.
  • Pettiford L; Division of Allergy and Immunology, Children's National Hospital, Washington, DC.
  • Kamani NR; Division of Allergy and Immunology, Children's National Hospital, Washington, DC.
  • Keller MD; Division of Allergy and Immunology, Children's National Hospital, Washington, DC.
  • Pham-Huy A; Division of Infectious Diseases, Immunology and Allergy, Children's Hospital Eastern Ontario, Ottawa, Ontario, Canada.
  • Grunebaum E; Division of Immunology and Allergy, The Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address: eyal.grunebaum@sickkids.ca.
J Allergy Clin Immunol Pract ; 11(6): 1725-1733, 2023 06.
Article en En | MEDLINE | ID: mdl-36736953
BACKGROUND: ADAGEN, a bovine-based enzyme replacement therapy (ERT), has been used to treat adenosine deaminase severe combined immunodeficiency (ADA-SCID). In 2018, ADAGEN was replaced by REVCOVI (elapegademase), a modified bovine recombinant protein. OBJECTIVE: To determine the real-life long-term benefits of REVCOVI in ADA-SCID. METHODS: Data on ERT, infectious and noninfectious complications, and metabolic and immune evaluations were collected from 17 patients with ADA-SCID treated for 6 months or more with REVCOVI. RESULTS: Eleven patients had previously received ADAGEN for 16 to 324 months, whereas 6 patients were ERT-naive. REVCOVI was administered twice weekly at 0.4 mg/kg/wk in ERT-naive patients, whereas patients transitioning to REVCOVI from ADAGEN typically continued at the same frequency and equivalent dosing as ADAGEN, resulting in a significantly lower (P = .007) total REVCOVI dose in the transitioning group. REVCOVI treatment in the ERT-naive group led to the resolution of many clinical and laboratory complications of ADA deficiency, whereas there were no new adverse effects among the transitioning patients. REVCOVI treatment increased plasma ADA activity and decreased dAXP (which included deoxyadenosine mono-, di-, and tri phosphate) among most patients, effects that persisted throughout the 7- to 37-month treatment periods, except in 2 patients with incomplete adherence. Among some patients, after 0.5 to 6 months, injection frequency was reduced to once a week, while maintaining adequate metabolic profiles. All ERT-naive infants treated with REVCOVI demonstrated an increase in the number of CD4+ T and CD19+ B cells, although these counts remained stable but lower than normal in most transitioning patients. CONCLUSIONS: REVCOVI is effective for the management of ADA-SCID.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inmunodeficiencia Combinada Grave / Reconstitución Inmune Límite: Animals / Humans / Infant Idioma: En Revista: J Allergy Clin Immunol Pract Año: 2023 Tipo del documento: Article País de afiliación: Canadá Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Inmunodeficiencia Combinada Grave / Reconstitución Inmune Límite: Animals / Humans / Infant Idioma: En Revista: J Allergy Clin Immunol Pract Año: 2023 Tipo del documento: Article País de afiliación: Canadá Pais de publicación: Estados Unidos