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Growth hormone improves short stature in children with Diamond-Blackfan anemia
Jonathan C Howell1, Sarita A Joshi, Lindsey Hornung
1Divisions of Endocrinology, Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children's Hospital Medical Center and University of Cincinnati, Cincinnati, Ohio.
Insights
Growth hormone (GH) therapy significantly improves growth velocity and height in children with Diamond-Blackfan anemia (DBA), a rare inherited condition. This treatment helps restore stature, offering a viable option for affected children.
Area of Science:
- Pediatric Endocrinology
- Hematology
- Genetics
Background:
- Diamond-Blackfan anemia (DBA) is an inherited bone marrow failure syndrome characterized by severe anemia and short stature in most patients.
- Previous isolated reports suggest growth hormone (GH) therapy may improve growth in children with DBA.
Purpose of the Study:
- To evaluate the efficacy of growth hormone (GH) therapy in improving growth parameters in children with Diamond-Blackfan anemia (DBA).
- To compare growth velocity (GV) and height-for-age Z-scores (HAZ) in GH-treated DBA patients versus non-GH-treated controls.
Main Methods:
- Collected GH treatment data from 19 children with DBA.
- Utilized control data from 44 non-GH treated children from the Diamond Blackfan Anemia Registry.
- Compared annual growth velocity (GV) and height-for-age Z-scores (HAZ) between groups and during up to 4 years of GH treatment.
Main Results:
- DBA-specific height charts revealed short stature compared to general population norms.
- GH-treated patients showed significantly lower baseline HAZ compared to controls.
- GH therapy led to significant improvements in GV within two years and HAZ annually over four years, achieving catch-up growth comparable to controls after two years.
Conclusions:
- Growth hormone (GH) treatment effectively improves growth velocity and height in children with Diamond-Blackfan anemia (DBA) for up to four years.
- GH therapy offers a successful strategy to restore stature in very short children with DBA.
- DBA-specific growth charts are valuable tools; individualized benefit-risk assessment for GH therapy is crucial given the associated malignancy risk.
Background:
Diamond-Blackfan anemia (DBA), an inherited marrow failure syndrome, has severe hypoplastic anemia in infancy and association with aplastic anemia, MDS/leukemia, and other malignancies. Short stature is present in most patients. Isolated cases have demonstrated improved growth on growth hormone (GH) therapy.
Procedures:
GH treatment data were obtained from 19 children with DBA (6 at our site and 13 from Genentech). Control data from 44 non-GH treated children were provided by Diamond Blackfan Anemia Registry. Annual growth velocity (GV) and height-for-age Z-scores (HAZ) were compared between groups and for up to 4y of GH treatment.
Results:
Constructed DBA-specific male and female height-for-age charts for non-GH treated patients revealed short stature compared to CDC norms. GH-treated patients had significantly lower HAZ prior to treatment initiation compared to non-GH-treated controls. Among GH-treated patients, GV significantly improved in the first two years relative to pre-treatment. HAZ significantly improved in each of 4y of GH therapy compared to baseline. After 2y of therapy, HAZ for GH-treated patients were not significantly different from controls, demonstrating successful catch-up growth.
Conclusions:
GH treatment in children with DBA improves both GV and HAZ during treatment sustained for up to 4y. Very short children with DBA can be treated successfully with GH to restore stature to levels comparable to less affected patients. DBA height charts are useful tools for assessing age-specific growth in this typically short population. Careful consideration of individualized benefit of GH therapy versus risk is important in view of long-term underlying ∼5% malignancy risk in DBA.
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