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Growth hormone therapy for short stature in Diamond Blackfan anemia

Emily Gale Scott1, Anzar Haider, Jeffrey Hord

  • 1Pediatric Emergency Medicine, Akron Children's Hospital, One Perkins Square, Akron, Ohio, USA. eg@neoucom.edu

Pediatric Blood & Cancer
|September 24, 2004
PubMed

Insights

Growth hormone (GH) therapy is beneficial for children with Diamond Blackfan anemia (DBA) experiencing short stature and slow growth. This case study shows improved growth velocity with daily GH injections.

Area of Science:

  • Pediatric Endocrinology
  • Hematology
  • Genetics

Background:

  • Diamond Blackfan anemia (DBA) is a rare inherited bone marrow failure syndrome.
  • Children with DBA often present with short stature and growth delay.
  • Growth hormone (GH) deficiency or resistance can contribute to poor growth in these patients.

Observation:

  • A 13-year-old male with DBA and short stature exhibited delayed bone age and low insulin-like growth factor 1 (IGF-1).
  • Despite a normal biochemical response to GH stimulation, his growth velocity was poor.
  • The patient was initiated on daily GH injections.

Findings:

  • Treatment with daily GH injections resulted in a significant increase in growth velocity, reaching 8.2 cm/year.
  • This indicates a positive therapeutic response to GH in this patient with DBA.

Implications:

  • Growth hormone therapy should be considered as a treatment option for children diagnosed with DBA who have short stature and poor growth velocity.
  • Further research may elucidate the mechanisms underlying GH response in DBA patients.
  • Optimizing growth in children with DBA can improve overall health outcomes.
Abstract

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