Long-term hydroxyurea therapy for infants with sickle cell anemia: the HUSOFT extension study

Abstract

The long-term efficacy and toxicity of hydroxyurea for infants are undefined, and its role in preventing organ dysfunction is unknown. Short-term feasibility of hydroxyurea administration, toxicities, hematologic effects, and effect on spleen function in infants with sickle cell anemia (

Evolution of sickle cell disease from a life‐threatening disease of children to a chronic disease of adults: The last 40 years

“…Shortly after the early phase hydroxyurea trials were completed in adults, several phase II trials established the tolerability and potential efficacy of hydroxyurea therapy in infants [52], toddlers [53], and older children [54]. The definitive benefits of hydroxyurea therapy in decreasing SCD related morbidity were replicated in children with hemoglobin SS (HbSS) or hemoglobin Sb(0)thalassaemia in a NHLBI sponsored study, the Hydroxyurea to Prevent Organ Damage in Children with Sickle Cell Anemia (BABY HUG) trial.…”

Outcome of hematopoietic cell transplantation in children with sickle cell disease, a single center's experience

“…[1][2][3][4] Although hydroxyurea therapy and chronic red blood cell transfusion may decrease the complications of chronic organ damage, 5,6 both treatment modalities require long-term compliance to prevent irreversible end-organ damage. Currently, however, hematopoietic stem cell transplantation (HCT) provides the only cure for SCD.…”

Fetal hemoglobin chemical inducers for treatment of hemoglobinopathies

“…Studies have been carried out in children with sickle cell anemia, showing the clinical efficacy and short/middle-term safety of HU [169][170][171][172]. In this context, particularly important were the two Hydroxyurea Safety and Organ Toxicity (HUSOFT) studies.…”

“…Hydroxyurea was shown also to be able to reduce the incidence of secondary stroke in children with sickle cell anemia and stroke [173]. The results of these two studies provided evidence that infants with sickle cell anemia tolerate HU therapy (up to 6 years of treatment) with sustained hematologic benefits, improved growth, and possibly preserved splenic function [169,170]. Importantly, the studies carried out in children showed that HU induces HbF levels to twice the average level achieved in adults [169][170][171][172].…”

“…The results of these two studies provided evidence that infants with sickle cell anemia tolerate HU therapy (up to 6 years of treatment) with sustained hematologic benefits, improved growth, and possibly preserved splenic function [169,170]. Importantly, the studies carried out in children showed that HU induces HbF levels to twice the average level achieved in adults [169][170][171][172]. Interestingly, in children with sickle cell anemia, higher baseline HbF predicted a better response to HU [172] and higher HbF levels under HU treatment were associated with a better hematological response [173].…”