Hydroxyurea Use in Young Children With Sickle Cell Anemia in New York State

Anders, David G.; Tang, Fei; Ledneva, Tatania; Casini, Michèle; Green, Nancy; Wang, Ying; Sturman, Lawrence S.

doi:10.1016/j.amepre.2016.01.001

Cited by 33 publications

(50 citation statements)

References 31 publications

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“…24,25 Future work should consider developmental screenings in SCD as an additional tool to identify children for preventative interventions. Children with positive screenings in this study were referred for followup services as part of the screening program, which could have decreased the predictive power for psychosocial outcomes.…”

Section: Discussionmentioning

confidence: 99%

Predictive validity of developmental screening in young children with sickle cell disease: a longitudinal follow‐up study

Schatz

Schlenz

Smith

et al. 2018

Develop Med Child Neuro

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Section: Discussionmentioning

confidence: 99%

Predictive validity of developmental screening in young children with sickle cell disease: a longitudinal follow‐up study

Schatz

Schlenz

Smith

et al. 2018

Develop Med Child Neuro

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“…5 Since that time, we have seen numerous reports demonstrating the safety and efficacy of HU in both children and adults with SCD. 3,[6][7][8][9][10][11][12][13][14][15][16] There is emerging evidence to support the use of HU in infants with SCD. Starting HU in infancy, before the expected drop in hemoglobin and hemoglobin F%, could lead to even better outcomes for our patients with SCD.…”

Section: Introductionmentioning

confidence: 99%

Hydroxyurea use in young infants with sickle cell disease

Schuchard

Lissick

Nickel

et al. 2019

Pediatric Blood & Cancer

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Background Hydroxyurea (HU) reduces complications and improves quality and duration of life in sickle cell disease. Evidence supports the use of HU starting after nine months of age. Procedures We performed a retrospective study of patients starting HU at less than five years of age between January 1, 2008, and December 31, 2016. We evaluated clinical events, laboratory data, and toxicity between three different age groups: cohort 1 (0–1 year), cohort 2 (1–2 years), and cohort 3 (2–5 years). Results Sixty‐five patients were included in the analysis. The mean age was 7.2 months (n = 35), 19.5 months (n = 13), and 35.5 months (n = 17) for cohorts 1, 2, and 3, respectively. Cohort 1 had higher hemoglobin (P = 0.0003) and MCV (P = 0.0199) and lower absolute reticulocyte count (P = 0.0304) at 24 months of age compared with cohort 3. The absolute neutrophil count (ANC) was lower compared with both older cohorts (P = 0.0364, 0.0025). The mean baseline hemoglobin F in cohort 1 was 31.5% compared with 19.7% and 16.5% in cohorts 2 and 3, respectively (P = 0.002, P < 0.0001). The mean duration of therapy was 31.3 months, 57.6 months (P = 0.018), and 29.1 months (P = 0.401), respectively. Mean Hb F levels remained higher in cohort 1 (29.9%) compared with cohorts 2 and 3 (20.4%, P = 0.007; 20.6%, P = 0.003). Cohort 1 experienced fewer hospitalizations (P = 0.0025), pain crises (P = 0.0618), and transfusions (P = 0.0426). There was no difference in toxicity between groups. Conclusion HU is safe and effective in patients 5 to 12 months of age and generated a more robust response compared with initiation in older patients.

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“…Recently published data from a cohort of 273 New York State Medicaid insured children born during 2006–2009 demonstrated that by 2014 approximately half were prescribed HU. While this is good news, HU treatment adherence of these youth is more sobering, as approximately two thirds of those treated met criteria for suboptimal adherence based on their pharmacy refill records [86]. Clearly interventions to improve HU adherence for youth with SCD are needed.…”

Section: Discussionmentioning

confidence: 99%

Study protocol for a randomized controlled trial to assess the feasibility of an open label intervention to improve hydroxyurea adherence in youth with sickle cell disease

Smaldone

Findley²,

Bakken

et al. 2016

Contemporary Clinical Trials

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Background Community health workers (CHW) are increasingly recognized as a strategy to improve health outcomes for the underserved with chronic diseases but has not been formally explored in adolescents with sickle cell disease (SCD). SCD primarily affects African American, Hispanic and other traditionally underserved populations. Hydroxyurea (HU), an oral, once-daily medication, is the only approved therapeutic drug for sickle cell disease and markedly reduces symptoms, morbidity and mortality and improves quality of life largely by increasing hemoglobin F blood levels. This paper presents the rationale, study design and protocol for an open label randomized controlled trial to improve parent-youth partnerships in self-management and medication adherence to HU in adolescents with SCD. Methods/Design A CHW intervention augmented by text messaging was designed for adolescents with SCD ages 10–18 years and their parents to improve daily HU adherence. Thirty adolescent parent dyads will be randomized with 2:1 intervention group allocation. Intervention dyads will establish a relationship with a culturally aligned CHW to identify barriers to HU use, identify cues to build a habit, and develop a dyad partnership to improve daily HU adherence and achieve their individualized “personal best” hemoglobin F target. Intervention feasibility, acceptability and efficacy will be assessed via a 2-site trial. Outcomes of interest are HU adherence, dyad self-management communication, quality of life, and resource use. Discussion Despite known benefits, poor HU adherence is common. If feasible and acceptable, the proposed intervention may improve health of underserved adolescents with SCD by enhancing long-term HU adherence.

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Hydroxyurea Use in Young Children With Sickle Cell Anemia in New York State

Cited by 33 publications

References 31 publications

Predictive validity of developmental screening in young children with sickle cell disease: a longitudinal follow‐up study

Predictive validity of developmental screening in young children with sickle cell disease: a longitudinal follow‐up study

Hydroxyurea use in young infants with sickle cell disease

Study protocol for a randomized controlled trial to assess the feasibility of an open label intervention to improve hydroxyurea adherence in youth with sickle cell disease

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