Five years of experience with hydroxyurea in children and young adults with sickle cell disease

Abstract

The short-term beneficial effect of hydroxyurea (HU) in sickle cell disease (SCD) has been proven by randomized studies in children and adults. The Belgian registry of HU-treated SCD patients was created to evaluate its long-term efficacy and toxicity. The median follow-up of the 93 patients registered is 3.5 years; clinical and laboratory data have been obtained for 82 patients at 1 year, 61 at 2 years, 44 at 3 years, 33 at 4 years, and 22 after 5 years. On HU, the number of hospitalizations and days hospitalized dropped significantly. Analysis of the 22 patients with a minimum of 5 years of follow-up confirm a significant difference in the number of hospitalizations (P ‫؍‬ .0002) and days in the hospital (P < .01), throughout the treatment when compared to prior to HU therapy. The probabilities of not experiencing any event or any vaso-occlusive crisis requiring hospitalization during the 5 years of treatment were, respectively, 47% and 55%. On HU, the rate per 100 patient-years of severe events was estimated to be 3.5% for acute chest syndrome, 1.2% for aplastic crisis, 0.4% for splenic sequestration; it was 0% for the 9 patients with a history of stroke or transient ischemic attack followed for an average of 4 years. No important adverse effect occurred. Long-term chronic treatment with HU for patients with SCD appears feasible, effective, and devoid of any major toxicity; in patients with a history of stroke, HU may be a valid alternative to chronic transfusion support. (Blood. 2001;97:3628-3632)

Hydroxyurea therapy for sickle cell disease in community‐based practices: A survey of Florida and North Carolina hematologists/oncologists

“…However, opinion experts have remained divided concerning the true leukemogenic potential of HU for this patient population [17,[20][21][22][23][24]; thus uncertainty remains today [25]. In a clinical trial of HU therapy for children and young adults with SCD, there were no cases of leukemia with a mean follow-up observation time of 3 years [26]. The literature addressing the risk of leukemia in SCD patients receiving HU was otherwise limited to two case reports [27,28].…”

Hidroxiuréia em pacientes com síndromes falciformes acompanhados no Hospital Hemope, Recife, Brasil

“…É bem tolerada, entre adultos, uma dose máxima de 2 g/dia, não sendo observados efeitos adversos, incluindo-se a mielossupressão, em experimentos de longo prazo. 15 A estes achados soma-se o efeito da hidroxiuréia sobre a O uso de HU em crianças e adultos portadores de SF parece ser seguro e eficaz e ainda pode promover melhora na qualidade de vida destes pacientes, benefícios a seus familiares, principalmente em regiões como o Nordeste do Brasil, carente de leitos e de pessoal especializado para seguimento dos mesmos.…”

Critical Reviews: How we treat sickle cell patients with leg ulcers

“…Moreover, there was no difference in ulcer prevalence (40%) between SCD patients that were taking or not taking hydroxyurea in a study of 505 patients at the NIH [5]. Several other authors are skeptical of a link between hydroxyurea and ulcers in SCD patients based on their own data [73][74][75], and many practical considerations preclude the randomized prospective trials needed for definitive evidence regarding this issue. There are many potential benefits of hydroxyurea use in SCD patients with ulcers: decrease in leukocyte count, decrease in inflammatory cytokines [76,77], increase in hemoglobin, increase in HbF, decrease in hemolysis, increase in oxygen carrying capacity, improved RBC rheology [78], and NO donor properties [79,80].…”