{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,22]],"date-time":"2025-10-22T02:47:51Z","timestamp":1761101271301},"reference-count":27,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2014,12,9]],"date-time":"2014-12-09T00:00:00Z","timestamp":1418083200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2015,3]]},"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>Diamond\u2010Blackfan 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.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedures<\/jats:title><jats:p>GH treatment data were obtained from 19 children with DBA (6 at our site and 13 from Genentech). Control data from 44 non\u2010GH treated children were provided by Diamond Blackfan Anemia Registry. Annual growth velocity (GV) and height\u2010for\u2010age Z\u2010scores (HAZ) were compared between groups and for up to 4y of GH treatment.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Constructed DBA\u2010specific male and female height\u2010for\u2010age charts for non\u2010GH treated patients revealed short stature compared to CDC norms. GH\u2010treated patients had significantly lower HAZ prior to treatment initiation compared to non\u2010GH\u2010treated controls. Among GH\u2010treated patients, GV significantly improved in the first two years relative to pre\u2010treatment. HAZ significantly improved in each of 4y of GH therapy compared to baseline. After 2y of therapy, HAZ for GH\u2010treated patients were not significantly different from controls, demonstrating successful catch\u2010up growth.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>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\u2010specific growth in this typically short population. Careful consideration of individualized benefit of GH therapy versus risk is important in view of long\u2010term underlying \u223c5% malignancy risk in DBA. Pediatr Blood Cancer 2015;62:402\u2013408. \u00a9 2014 Wiley Periodicals, Inc.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.25341","type":"journal-article","created":{"date-parts":[[2014,12,9]],"date-time":"2014-12-09T19:03:57Z","timestamp":1418151837000},"page":"402-408","source":"Crossref","is-referenced-by-count":10,"title":["Growth hormone improves short stature in children with Diamond\u2010Blackfan anemia"],"prefix":"10.1002","volume":"62","author":[{"given":"Jonathan C.","family":"Howell","sequence":"first","affiliation":[{"name":"Divisions of Endocrinology Bone Marrow Transplantation and Immune Deficiency Department of Pediatrics Cincinnati Children's Hospital Medical Center and University of Cincinnati Cincinnati Ohio"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sarita A.","family":"Joshi","sequence":"additional","affiliation":[{"name":"Division of Hematology\/Oncology and BMT Department of Pediatrics Nationwide Children's Hospital Columbus 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