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Published on: January 7, 2016
Use of recombinant human growth hormone in children with thalassemia
Mitchell E Geffner1, Hanna Karlsson
1Saban Research Institute, Childrens Hospital Los Angeles, Keck School of Medicine, University of Southern California, Los Angeles, Calif., USA. mgeffner@chla.usc.edu
Insights
Recombinant human growth hormone (rhGH) effectively increases growth rates in short children with thalassemia. However, growth outcomes in those without growth hormone deficiency (GHD) require further investigation.
Area of Science:
- Pediatrics
- Endocrinology
- Hematology
Background:
- Chronic anemias, particularly thalassemia, are associated with growth failure in children.
- Approximately 30% of children with thalassemia experience growth impairment.
Purpose of the Study:
- To assess the efficacy of recombinant human growth hormone (rhGH) in improving growth in children with thalassemia.
- To evaluate growth hormone (GH) status in short children with thalassemia.
Main Methods:
- Analysis of a cohort of 147 short children with thalassemia from the Pfizer International Growth Study Database (KIGS).
- Assessment of treatment with rhGH for one year.
- Evaluation of baseline characteristics including GH deficiency (GHD), birth weight, parental height, and insulin-like growth factor I (IGF-I) levels.
Main Results:
- Approximately 40% of short thalassemic children in the study had GHD.
- rhGH treatment significantly increased growth rates in thalassemic children compared to baseline.
- Growth rates achieved with rhGH were lower than those in short children with or without GHD.
Conclusions:
- GH testing is recommended for short children with thalassemia.
- rhGH treatment is indicated for thalassemic children diagnosed with GHD.
- The benefit of rhGH therapy for short thalassemic children without GHD needs further study, particularly regarding final height.
Background:
Growth failure occurs in children with chronic anemias and, in particular, in approximately 30% of those with thalassemia.
Methods:
We assessed recombinant human growth hormone (rhGH) use in a large cohort of children with thalassemia enrolled in the Pfizer International Growth Study Database (KIGS).
Results:
We identified 147 short children with thalassemia who were treated with rhGH in KIGS. Of these, approximately 40% had a primary diagnosis of GH deficiency (GHD). They had low birth weight, short parents, reduced genetic height potential, low insulin-like growth factor I levels and delayed bone age. Treatment with rhGH for 1 year resulted in a significantly increased growth rate regardless of underlying GH or pubertal status. Although the resultant growth rates for thalassemic children were significantly higher than at baseline, they were less than those seen in similarly treated short children with or without GHD.
Conclusions:
GH testing should be performed in short thalassemic children, and those with GHD should be treated with rhGH. The value of rhGH therapy in short thalassemic children without GHD is less clear-cut and requires further study regarding final height outcome.
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