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Managing the child with severe primary insulin-like growth factor-1 deficiency (IGFD): IGFD diagnosis and management

Jay Cohen1, Sandra Blethen, Joyce Kuntze

  • 1BMG: The Endocrine Clinic, P.C., 5659 South Rex Road, Memphis, TN, 38119, USA, jaycohen1a@icloud.com.

Drugs in R&D
|March 19, 2014
PubMed

Insights

Severe primary insulin-like growth factor 1 (IGF-1) deficiency (SPIGFD) in children can be effectively treated with mecasermin. This review details safe and effective mecasermin use, including dosing, side effects, and reinitiation after drug shortages.

Area of Science:

  • Pediatric Endocrinology
  • Growth Hormone Therapy
  • Metabolic Disorders

Background:

  • Severe primary insulin-like growth factor 1 (IGF-1) deficiency (SPIGFD) causes significant growth failure.
  • SPIGFD is characterized by low IGF-1 and height standard deviation scores (SDS) with normal/high growth hormone.
  • Mecasermin, a recombinant human IGF-1, is a treatment option for SPIGFD.

Purpose of the Study:

  • To provide guidance on the safe and effective use of mecasermin for SPIGFD.
  • To outline treatment initiation, monitoring, and management of side effects.
  • To address reinitiation of mecasermin therapy following drug shortages.

Main Methods:

  • Review of current literature and clinical guidelines on mecasermin therapy.
  • Description of dosing strategies for mecasermin in pediatric patients.
  • Identification and discussion of common and significant side effects.

Main Results:

  • Mecasermin treatment can successfully improve growth in children with SPIGFD.
  • Careful monitoring of IGF-1 levels and side effects is crucial for safe treatment.
  • Established protocols exist for initiating and managing mecasermin therapy.

Conclusions:

  • Mecasermin is a valuable therapeutic agent for SPIGFD, improving growth outcomes.
  • Adherence to recommended dosing and monitoring practices ensures patient safety.
  • Strategies for managing treatment interruptions and reinitiation are essential, especially considering potential drug shortages.

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