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Eculizumab in Adolescent Patients With Refractory Generalized Myasthenia Gravis: A Phase 3, Open-Label, Multicenter Study.
Brandsema, John F; Ginsberg, Matthew; Hoshino, Hideki; Mimaki, Masakazu; Nagata, Satoru; Rao, Vamshi K; Ruzhansky, Katherine; Suresh, Niraja; Tiongson, Emmanuelle; Yamanouchi, Hideo; Frick, Glen; Hicks, Eden; Liao, Serena; Howard, James F.
Affiliation
  • Brandsema JF; Division of Neurology, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
  • Ginsberg M; Department of Neurology, Akron Children's Hospital, Akron, Ohio.
  • Hoshino H; Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan.
  • Mimaki M; Department of Pediatrics, Teikyo University School of Medicine, Tokyo, Japan.
  • Nagata S; Department of Pediatrics, Tokyo Women's Medical University School of Medicine, Tokyo, Japan.
  • Rao VK; Division of Neurology, Lurie Children's Hospital, Department of Pediatrics, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
  • Ruzhansky K; Department of Neurology, Medical University of South Carolina, Charleston, South Carolina.
  • Suresh N; Department of Neurology, University of South Florida, Tampa, Florida.
  • Tiongson E; Division of Neurology, Children's Hospital Los Angeles, Los Angeles, California.
  • Yamanouchi H; Department of Pediatrics, Comprehensive Epilepsy Center, Saitama Medical University, Saitama, Japan.
  • Frick G; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts. Electronic address: Glen.Frick@alexion.com.
  • Hicks E; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts.
  • Liao S; Alexion, AstraZeneca Rare Disease, Boston, Massachusetts.
  • Howard JF; Department of Neurology, The University of North Carolina, Chapel Hill, North Carolina.
Pediatr Neurol ; 156: 198-207, 2024 Jul.
Article in En | MEDLINE | ID: mdl-38810600
ABSTRACT

BACKGROUND:

This study evaluated the efficacy and safety of eculizumab, a terminal complement C5 inhibitor, in juvenile generalized myasthenia gravis (gMG).

METHODS:

Adolescents aged 12 to 17 years with refractory anti-acetylcholine receptor (AChR) antibody-positive gMG received eculizumab (weekly induction [one to two doses of 600 mg or four doses of 900 mg] followed by maintenance doses [300 to 1200 mg] every two weeks for up to 26 weeks) in a phase 3, open-label multicenter study (NCT03759366). Change from baseline to week 26 in Quantitative Myasthenia Gravis (QMG) total score (primary end point) and secondary end points including Myasthenia Gravis-Activities of Daily Living (MG-ADL) total score, Myasthenia Gravis Composite score, Myasthenia Gravis Foundation of America postintervention status, EuroQol 5-Dimensions (Youth) and Neurological Quality-of-Life Pediatric Fatigue questionnaire scores, as well as pharmacokinetics, pharmacodynamics, and safety, were recorded.

RESULTS:

Eleven adolescents (mean ± S.D. age 14.8 ± 1.8 years) were enrolled; 10 completed the primary evaluation period. Least-squares mean changes from baseline at week 26 were -5.8 (standard error [SE] 1.2; P = 0.0004) for QMG total score and -2.3 (SE 0.6; P = 0.0017) for MG-ADL total score. Overall, the primary and all secondary efficacy end point analyses met statistical significance from the first assessment and were sustained throughout. Complete terminal complement inhibition was sustained through 26 weeks in all patients. Treatment-emergent adverse events were all mild/moderate and predominantly unrelated to eculizumab.

CONCLUSIONS:

Eculizumab was effective in reducing disease burden and was well tolerated in adolescents with refractory AChR antibody-positive gMG.
Subject(s)
Key words

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Complement Inactivating Agents / Antibodies, Monoclonal, Humanized / Myasthenia Gravis Limits: Adolescent / Child / Female / Humans / Male Language: En Journal: Pediatr Neurol Journal subject: NEUROLOGIA / PEDIATRIA Year: 2024 Document type: Article

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Complement Inactivating Agents / Antibodies, Monoclonal, Humanized / Myasthenia Gravis Limits: Adolescent / Child / Female / Humans / Male Language: En Journal: Pediatr Neurol Journal subject: NEUROLOGIA / PEDIATRIA Year: 2024 Document type: Article