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United States clinical practice experience with eculizumab in myasthenia gravis: symptoms, function, and immunosuppressant therapy use.
Habib, Ali A; Klink, Andrew J; Muppidi, Srikanth; Parthan, Anju; Sader, S Chloe; Balanean, Alexandrina; Gajra, Ajeet; Nowak, Richard J; Howard, James F.
Afiliação
  • Habib AA; University of California, Irvine, CA, USA.
  • Klink AJ; Cardinal Health, Dublin, OH, USA.
  • Muppidi S; Stanford Neuroscience Health Center, Palo Alto, CA, USA.
  • Parthan A; Alexion, AstraZeneca Rare Disease, Boston, MA, USA.
  • Sader SC; FibroGen Inc., San Francisco, CA, USA.
  • Balanean A; Alexion, AstraZeneca Rare Disease, Boston, MA, USA.
  • Gajra A; Cardinal Health, Dublin, OH, USA.
  • Nowak RJ; Cardinal Health, Dublin, OH, USA.
  • Howard JF; Hematology-Oncology Associates of CNY, East Syracuse, NY, USA.
J Neurol ; 271(9): 6114-6126, 2024 Sep.
Article em En | MEDLINE | ID: mdl-39052039
ABSTRACT
BACKGROUND/

OBJECTIVES:

The phase 3 REGAIN study and its open-label extension demonstrated the efficacy of the complement C5 inhibitor eculizumab in patients with treatment-refractory, acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG). The aim of the ELEVATE study was to assess the effectiveness of eculizumab in clinical practice in adults with MG in the United States.

METHODS:

A retrospective chart review was conducted in adults with MG who initiated eculizumab treatment between October 23, 2017 and December 31, 2019. Outcomes assessed before and during eculizumab treatment using a pre- versus post-treatment study design included Myasthenia Gravis-Activities of Daily Living (MG-ADL) total scores; minimal symptom expression (MSE); physician impression of clinical change; minimal manifestation status (MMS); and concomitant medication use.

RESULTS:

In total, 119 patients were included in the study. A significant reduction was observed in mean MG-ADL total score, from 8.0 before eculizumab initiation to 5.4 at 3 months and to 4.7 at 24 months after eculizumab initiation (both p < 0.001). At 24 months after eculizumab initiation, MSE was achieved by 19% of patients. MMS or better was achieved by 30% of patients at 24 months. Additionally, 64% of patients receiving prednisone at eculizumab initiation had their prednisone dosage reduced during eculizumab treatment and 13% discontinued prednisone; 32% were able to discontinue nonsteroidal immunosuppressant therapy.

DISCUSSION:

Eculizumab treatment was associated with sustained improvements in MG-ADL total scores through 24 months in adults with MG. Prednisone dosage was reduced in approximately two-thirds of patients, suggesting a steroid-sparing effect for eculizumab.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Inativadores do Complemento / Anticorpos Monoclonais Humanizados / Imunossupressores / Miastenia Gravis Limite: Adult / Aged / Female / Humans / Male / Middle aged País/Região como assunto: America do norte Idioma: En Revista: J Neurol Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Alemanha
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Inativadores do Complemento / Anticorpos Monoclonais Humanizados / Imunossupressores / Miastenia Gravis Limite: Adult / Aged / Female / Humans / Male / Middle aged País/Região como assunto: America do norte Idioma: En Revista: J Neurol Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos País de publicação: Alemanha