Safety and efficacy of dose‐escalation hydroxyurea therapy in very young children with sickle cell anemia: A retrospective cohort study

2020 ◽  
Vol 67 (9) ◽  
Author(s):  
Alex George ◽  
Jennifer N. Tran
Keyword(s):  
Cohort Study ◽  
Young Children ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Dose Escalation ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Safety And Efficacy ◽  
Very Young Children ◽  
Hydroxyurea Therapy

Escalating Doses Of Hydroxyurea In Very Young Children With Sickle Cell Anemia

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 978-978
Author(s):  
Shangli Lian ◽  
Jeremie H. Estepp ◽  
Matthew P. Smeltzer ◽  
Winfred C. Wang
Keyword(s):  
Young Children ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Dose Escalation ◽  
Fixed Dose ◽  
Early Therapy ◽  
Hematologic Response ◽  
Very Young Children ◽  
The Impact

Abstract Background In the HUSOFT and BABY HUG trials (Wang, et al., J. Pediat, 2001 and Lancet, 2011), very young children (6-24 and 9-18 months old at entry, respectively) with sickle cell anemia (SCA) initiated hydroxyurea (HU) at a fixed dose of 20 mg/kg/day, which improved hematologic parameters, provided substantial clinical benefits, and had an excellent safety profile. These results encouraged parental acceptance of HU treatment in very young children with SCA and in some parents promoted an expectation of early therapy. At our institution, select children as young as 6 months of age have initiated HU upon parental request. As in HUSOFT and BABY HUG, these children have begun at a dose of ∼20 mg/kg/day but then had dose escalation, based on hematologic parameters, toward a maximal tolerated dose (Ware, et al., Blood, 2010). Because the impact of dose escalation in this population is unknown, we have retrospectively reviewed our experience. Methods Children with homozygous sickle cell anemia (HbSS) who began HU therapy prior to 18 months of age between June 2010 and October 2012 were retrospectively identified following IRB approval. Demographic data, dosage information, and laboratory results pre-HU and at last clinical follow-up were analyzed. Descriptive data are presented below as means (±SD) unless otherwise noted. Statistical comparisons were performed with the Exact Wilcoxon Signed Rank test. Results Participants (n=6; female=3) were begun on HU because of recurrent dactylitis (n=2) or parental request (n=4). The mean initial dose of HU was 19.1 (±1.6) mg/kg/d, which subsequently was escalated to 25.4 (±4.4) mg/kg/d over an average of 14.8 (±10.5; range, 4.9-32.1) months. HU therapy was interrupted briefly in 2 participants for neutropenia and reticulocytopenia, respectively. Both incidents were felt related to viral suppression and both participants resumed HU at their previous dosage without further toxicities. Hematologic response is summarized in the following table along with hematologic response data reported in the HUSOFT and BABY HUG trials in subjects of comparable age. Discussion Six patients with HbSS who were begun on treatment at a mean age of 12 months had their dose of hydroxyurea escalated over an average follow-up period of 15 months. Hematologic responses included marked increases in Hb level, HbF, and MCV and a decrease in ARC, along with relatively stable neutrophil and platelet counts. When compared with subjects of approximately the same age in the BABY HUG and HUSOFT trials, all of whom were treated at doses of ≤20 mg/kg/d, our patients may have demonstrated more robust changes in their Hb, HbF, MCV and ARC levels, possibly related to their higher hydroxyurea dosing. The ongoing BABY HUG follow-up studies should provide complementary information for subjects who are ≥3 years of age, since they are having dose escalation toward maximum tolerated dose (MTD) and will be evaluated over an extended period of time. Overall, our current data suggest that increasing hydroxyurea dosage starting at a very early age is safe and hematologically efficacious and warrants further exploration with the goal of maximizing clinical benefit. Disclosures: Off Label Use: Hydroxurea therapy in children with sickle cell disease.

scholarly journals Clinical features and outcome of sickle cell anemia in a tertiary center: A retrospective cohort study

2018 ◽  
Vol 9 (1) ◽  
pp. 22
Author(s):  
MohsenSaadi Alzahrani ◽  
AbdulrahmanMusaad Alhumaid ◽  
AbdulmalekSuliman Aleidi ◽  
AbdullelahSaleh Alfakhri ◽  
NaifKhalil Alosaimi ◽  
...  
Keyword(s):  
Cohort Study ◽  
Clinical Features ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Tertiary Center

Safety of Deep Sedation in Young Children with Sickle Cell Disease: A Retrospective Cohort Study

2015 ◽  
Vol 166 (5) ◽  
pp. 1226-1232 ◽  
Author(s):  
Ami P. Belmont ◽  
Fadi Nossair ◽  
Donald Brambilla ◽  
Marjorie Friedman ◽  
Jan Boswinkel ◽  
...  
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Young Children ◽  
Sickle Cell ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Deep Sedation ◽  
Cell Disease

Costs Associated with the Care of Very Young Children with Sickle Cell Anemia (SCA): Analysis from the BABY HUG Study

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 171-171
Author(s):  
Winfred C. Wang ◽  
Suzette O. Oyeku ◽  
Zhaoyu Luo ◽  
Sheree L. Boulet ◽  
Scott T. Miller ◽  
...  
Keyword(s):  
Young Children ◽  
Sickle Cell ◽  
Sickle Cell Anemia ◽  
Inpatient Cost ◽  
Acute Chest Syndrome ◽  
Costs Of Care ◽  
Very Young Children ◽  
Thomson Reuters ◽  
Hydroxyurea Treatment ◽  
Hydroxyurea Therapy

Abstract Abstract 171 Background: The BABY HUG trial was a multi-center double-blinded randomized comparison of hydroxyurea (HU) versus placebo in infants (mean age 13.6 mo. at entry) with HbSS/Sβ° thalassemia who were followed for 2 years [Lancet 377(2011):1663–1672; Clinical Trials #NCT00006400]. Hydroxyurea therapy was associated with less pain, dactylitis, acute chest syndrome (ACS), hospitalization (HSN), and transfusion (TX) and with improved hematologic values; toxicity was limited to mild-moderate neutropenia. On the basis of the safety and efficacy data from this trial, it was concluded that hydroxyurea therapy can be considered for all very young children with sickle cell anemia (SCA). With anticipated broader use of hydroxyurea in this population, we examined estimated medical costs of care (based on Medicaid reimbursement) in treated versus placebo subjects. Methods: The BABY HUG database (C-TASC, Baltimore, MD) was utilized to compare inpatient events in subjects receiving hydroxyurea with those receiving placebo. Unit costs were estimated from the 2009 Thomson Reuters MarketScan® Multi-state Medicaid Database for children with HbSS (ICD-9 codes 282.61 or 282.62), ages 1–3 years. Inpatient costs included emergency department (ED) costs for admissions from an ED in about 80% of the 748 admissions in the database. Inpatient cost estimates were based on length of stay (LOS) modified by a diagnosis of ACS or splenic sequestration (SpS) or a procedure code for a TX. Outpatient expenses were estimated based on the schedule required for BABY HUG (and recommended for clinical use) and a “standard” schedule for 1–3 year-olds with SCA based on management protocols at 3 pediatric sickle cell centers in the US. Results: 96 subjects were randomized to hydroxyurea (83 completed the trial); 97 received placebo (84 completed the trial). In the full study, there were 232 hospitalizations (for any cause) in those receiving hydroxyurea and 324 in those on placebo; inpatient data were captured for only the final 77% of admissions (between 2/06 and 9/09). The LOS for subjects receiving hydroxyurea (mean 3.7, median 3, range 1–9 days) did not differ from those receiving placebo (3.6, 3, 1–13). Estimated inpatient and outpatient costs are shown in Tables 1 and 2. When inpatient and outpatient expenses were combined, the annual cost for 1–3 year-old children with SCA was $11,345 on hydroxyurea and $14,815 on placebo, a difference of $3,470. Discussion/Conclusion: Despite increased outpatient care expenses from clinic visits, laboratory monitoring, and hydroxyurea, savings on inpatient care resulted in an overall reduction in estimated annual per patient expenditure of approximately 23%. A limitation of our analysis was the dependence on MarketScan Medicaid data in lieu of the availability of specific expenses of the subjects participating in the BABY HUG study. Medicaid data may understate costs of care; based on prior analyses, we estimate that costs to private payers may be 20–30% greater than Medicaid reimbursements. We conclude that increased use of hydroxyurea treatment in children with SCA can lead to significant medical cost savings. Disclosures: Off Label Use: Hydroxyurea is not indicated for treatment of children with sickle cell disease. Use of this medication was for clinical indications and not mandated by this observational study.

Retrospective Cohort Study Comparing the Effect of Obesity in Children with Sickle Cell Disease

PEDIATRICS ◽  
2016 ◽  
Vol 137 (Supplement 3) ◽  
pp. 264A-264A
Author(s):  
Parth J. Bhatt ◽  
Dinesh Singh ◽  
Akingbola Olubenga ◽  
Devraj Chavda ◽  
Achint Patel
Keyword(s):  
Cohort Study ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Cell Disease ◽  
Obesity In Children
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