A clinically meaningful fetal hemoglobin threshold for children with sickle cell anemia during hydroxyurea therapy
| Autor: | Russell E. Ware, Jane S. Hankins, Kerri Nottage, Christina M. Abrams, Guolian Kang, Banu Aygun, Chen Li, Winfred C. Wang, Jeremie H. Estepp, Matthew P. Smeltzer |
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| Rok vydání: | 2017 |
| Předmět: |
Male
congenital hereditary and neonatal diseases and abnormalities Pediatrics medicine.medical_specialty Adolescent Maximum Tolerated Dose Anemia Anemia Sickle Cell Article 03 medical and health sciences 0302 clinical medicine Antisickling Agents hemic and lymphatic diseases Fetal hemoglobin Humans Hydroxyurea Medicine Prospective Studies Dosing Child Prospective cohort study Fetal Hemoglobin Dose-Response Relationship Drug business.industry Infant Hematology medicine.disease Sickle cell anemia Blood Cell Count Hospitalization Red blood cell medicine.anatomical_structure Child Preschool 030220 oncology & carcinogenesis Toxicity Female Observational study business 030215 immunology |
| Zdroj: | American Journal of Hematology. 92:1333-1339 |
| ISSN: | 0361-8609 |
| Popis: | Hydroxyurea has proven clinical benefits and is recommended to be offered to all children with sickle cell anemia (SCA), but the optimal dosing regimen remains controversial. Induction of red blood cell fetal hemoglobin (HbF) by hydroxyurea appears to be dose-dependent. However, it is unknown whether maximizing HbF% improves clinical outcomes. HUSTLE (NCT00305175) is a prospective observational study with a primary goal of describing the long-term clinical effects of hydroxyurea escalated to maximal tolerated dose (MTD) in children with SCA. In 230 children, providing 610 patient-years of follow up, the mean attained HbF% at MTD was >20% for up to 4 years of follow-up. When HbF% values were ≤20%, children had twice the odds of hospitalization for any reason (p20% was associated with fewer hospitalizations without significant toxicity. These data support the use of hydroxyurea in children, and suggest that the preferred dosing strategy is one that targets a HbF endpoint >20%. |
| Databáze: | OpenAIRE |
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