Long-Term Immune Reconstitution in ADA-Deficient Patients Treated With Elapegademase: A Real-World Experience.

Infant Humans Animals Cattle Adenosine Deaminase / therapeutic use Immune Reconstitution Severe Combined Immunodeficiency / therapy
Adenosine deaminase deficiency Enzyme replacement Polyethylene glycol Severe combined immunodeficiency

Journal

The journal of allergy and clinical immunology. In practice
ISSN: 2213-2201
Titre abrégé: J Allergy Clin Immunol Pract
Pays: United States
ID NLM: 101597220

Informations de publication

Date de publication:
06 2023
Historique:
received: 03 10 2022
revised: 06 01 2023
accepted: 09 01 2023
medline: 12 6 2023
pubmed: 4 2 2023
entrez: 3 2 2023
Statut: ppublish

Résumé

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. To determine the real-life long-term benefits of REVCOVI in ADA-SCID. 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. 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 REVCOVI is effective for the management of ADA-SCID.

Sections du résumé

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
CONCLUSIONS
REVCOVI is effective for the management of ADA-SCID.

Identifiants

DOI: 10.1016/j.jaip.2023.01.028 PMID: 36736953
pubmed: 36736953
pii: S2213-2198(23)00114-9
doi: 10.1016/j.jaip.2023.01.028
pii:
doi:

Substances chimiques

elapegademase 9R3D3Y0UHS
Adenosine Deaminase EC 3.5.4.4

Types de publication

Journal Article Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1725-1733

Informations de copyright

Copyright © 2023 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.

Auteurs

Luis Murguia-Favela (L)

Section of Hematology/Immunology, Department of Pediatrics, Alberta Children's Hospital and the University of Calgary, Calgary, Alberta, Canada.

Sneha Suresh (S)

Division of IHOPE, Department of Pediatrics, University of Alberta, Stollery Children's Hospital, Edmonton, Alberta, Canada.

Nicola A M Wright (NAM)

Section of Hematology/Immunology, Department of Pediatrics, Alberta Children's Hospital and the University of Calgary, Calgary, Alberta, Canada.

Saima Alvi (S)

Division of Pediatric Hematology/Oncology, Jim Pattison Children's Hospital, Saskatoon, Saskatchewan, Canada.

Sarah Tehseen (S)

Division of Hematology/Oncology and Transfusion Medicine, Jim Pattison Children's Hospital, Saskatoon, Saskatchewan, Canada.

Vivian Hernandez-Trujillo (V)

Allergy and Immunology Care Center of South Florida, Miami Lakes, Fla.

Christine M Seroogy (CM)

Division of Allergy, Immunology & Rheumatology, University of Wisconsin School of Medicine and Public Health, Madison, Wis.

Elie Haddad (E)

Division of Immunology and Rheumatology, Department of Pediatrics, Centre Hospitalier Universitaire (CHU) Sainte-Justine, University of Montreal, Montreal, Quebec, Canada.

Daime Nieves (D)

Division of Pediatric Allergy and Immunology, University of South Florida at Johns Hopkins All Children's Hospital, St Petersburg, Fla.

Michael S Hershfield (MS)

Division of Medicine and Biochemistry, Duke University Medical Center, Durham, NC.

Jolan E Walter (JE)

Division of Pediatric Allergy and Immunology, University of South Florida at Johns Hopkins All Children's Hospital, St Petersburg, Fla.

Leah Pettiford (L)

Division of Allergy and Immunology, Children's National Hospital, Washington, DC.

Naynesh R Kamani (NR)

Division of Allergy and Immunology, Children's National Hospital, Washington, DC.

Michael D Keller (MD)

Division of Allergy and Immunology, Children's National Hospital, Washington, DC.

Anne Pham-Huy (A)

Division of Infectious Diseases, Immunology and Allergy, Children's Hospital Eastern Ontario, Ottawa, Ontario, Canada.

Eyal Grunebaum (E)

Division of Immunology and Allergy, The Hospital for Sick Children, Toronto, Ontario, Canada. Electronic address: eyal.grunebaum@sickkids.ca.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Nourrisson › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Eucaryotes › Animaux › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Artiodactyla › Ruminants › Bovins › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Enzymes et coenzymes › Enzymes › Hydrolases › Aminohydrolases › Nucleoside deaminases › Adenosine deaminase › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Phénomènes du système immunitaire › Reconstitution immunitaire › Long-Term Immune Reconstitution in...
questionsmedicales.fr › Maladies du système immunitaire › Déficits immunitaires › Maladies d'immunodéficience primaire › Immunodéficience combinée grave › Long-Term Immune Reconstitution in...