Cost-effectiveness of adenotonsillectomy in reducing obstructive sleep apnea, cerebrovascular ischemia, vaso-occlusive pain, and ACS episodes in pediatric sickle cell disease.

In children with sickle cell disease (SCD), adenotonsillar hypertrophy or recurrent tonsillitis are frequently linked with an increased risk of obstructive sleep apnea, cerebrovascular ischemia, or frequent pain episodes and often require an adenoidectomy and/or tonsillectomy. Interventions designed to prevent these complications, control vaso-occlusive pain episodes, and avoid hospitalizations may reduce the significant personal and economic burden of SCD. This study compares episode recurrence and treatment costs for cerebrovascular ischemia, vaso-occlusive pain, acute chest syndrome (ACS), and obstructive sleep apnea in children who had an adenotonsillectomy (A/T surgery, N = 256; 11.7%) and a matched cohort of those who did not (N = 512; 23.3%) from a cohort of 2,194 children and adolescents with SCD from South Carolina's Medicaid system. A/T surgery was associated with a significantly reduced rate of visits over time for obstructive sleep apnea and cerebrovascular ischemia (e.g., stroke, transient ischemic attacks), but not with any change in the rate of visits for vaso-occlusive pain or ACS/pneumonia visits. The rate of mean acute (emergency and inpatient) service costs was significantly decreasing over time after an increase about the time the A/T surgery was performed. The cost-effectiveness of adenoidectomy and/or tonsillectomy for treating obstructive sleep apnea and preventing cerebrovascular ischemia without increasing vaso-occlusive pain episodes or long-term acute service costs in routine clinical practice settings was demonstrated. The matched control group of SCD patients without A/T surgery contained more patients with severe vaso-occlusive pain episodes, ACS visits, and higher mean total costs over time and appears to represent a different phenotype of children with SCD.

Pain management in children and adolescents with sickle cell disease.

In a cohort of 2,194 children with sickle cell disease (SCD) treated in community-based services, we explored the types of medications used to treat vaso-occlusive (VOC) pain episodes, and the relative effectiveness of nonsteroidal anti-inflammatory drugs (NSAIDs), opioids, and adjunctive antidepressants or anticonvulsant medications on reducing acute VOC pain visits over time. Pharmacologic treatments for VOC pain consisted mainly of NSAIDs and weak opioids. Significantly more patients with more than 3 inpatient or ER VOC pain visits during their first year of SCD treatment were prescribed stronger opioids, SSRIs, SNRI/heterocyclics, and anticonvulsants. Prescription of both stronger opioids and SSRI antidepressants or anticonvulsants was significantly associated with lower cumulative rates of acute VOC pain visits over time. Using an observational study design and existing clinical data, these findings are intended to illustrate the potential clinical advantages of combining adjunctive antidepressants or anticonvulsants with primary pain medications for relief of acute VOC pain over time.

Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea.

BACKGROUND: More evidence of the safety and effectiveness of hydroxyurea (HU) in community-based cohorts of pediatric patients with sickle cell disease (SCD) are needed. The association of HU with organ-specific clinical complications and adverse events is examined herein. METHODS: Medicaid medical and pharmacy claims for the calendar years January 1996 through December 2006 were used to identify a cohort of children and adolescent patients (ages 17 and under) with a diagnosis of SCD (homozygous) who were treated with HU and developed disparate complications or adverse side effects. Of the 2,194 pediatric SCD patients identified, 175 (8%) were treated with HU. Incidence density matching (1 case: 2 controls) was used to select the control group on age, gender, ethnicity, time in the Medicaid data set, and baseline severity resulting in a total study cohort of 523 cases. RESULTS: Organ-specific complications were more likely in the HU-treated group compared to non-HU-treated group: cardiovascular complications (odds ratio [OR] = 3.15; confidence interval [CI] = 1.97-5.03); hepatic complications (OR 5.41; CI = 3.54-8.27); renal complications (OR 5.09; CI 3.37-7.67); and pulmonary complications (OR 4.07; CI 1.88-8.79). Many of these conditions began developing before HU was prescribed. Developing three or more complications was also more likely in the HU group (27.4% vs. 7.0%, P < 0.0001). CONCLUSIONS: Extending previous findings to routine practice settings, HU is being administered to the most severely ill children with SCD, many of whom had already started to develop organ-specific complications, but it is not associated with development of serious adverse events.

Prevalence, treatment, and outcomes of renal conditions in pediatric sickle cell disease.

OBJECTIVES: Vaso-occlusive events in pediatric sickle cell disease (SCD) may cause various renal complications and lead to renal failure. We describe the renal conditions that develop among young patients with SCD and the factors associated with the prevalence of these nephropathies. MATERIALS AND METHODS: Medicaid medical and pharmacy claims for an 11-year period were used to identify 2194 pediatric patients with SCD (HbSS homozygous). Survival analysis identified the most significant predictors of acute kidney injury and chronic renal failure, using demographics, SCD severity and pain medication, comorbid hypertension, hematuria, and proteinuria as the initial covariates. RESULTS: Prevalence of renal complications in our cohort was found to be relatively low, predominantly hematuria (6.3%) and proteinuria (3.2%). The multivariable analysis indicated that earlier development of acute kidney injury was significantly associated with older age (adjusted hazard ratio [aHR] 1.16, confidence interval [CI] 1.06-1.27), preexisting hypertension (aHR 3.05, CI 1.09-8.60), and preexisting hematuria (aHR 2.87, CI 1.05-7.93). Earlier development of chronic renal failure was significantly associated with older age (aHR 1.20, CI 1.08-1.32), preexisting hematuria (aHR 4.67, CI 1.57-13.94), and preexisting proteinuria (aHR 8.25, CI 2.12-10.38). CONCLUSIONS: These prevalence findings are novel in the US SCD pediatric population. The predictors of nephropathies identified in these children confirm clinical expectations. In addition, they suggest not only that pediatric nephrologists should be consulted earlier in the treatment of patients with SCD who are diagnosed as having comorbid hypertension or who develop hematuria or proteinuria during the course of their SCD treatment but also that both hydroxyurea and angiotensin-converting enzyme inhibitor therapies may be better used in these cases.

Cost-effectiveness of hydroxyurea in reducing the frequency of pain episodes and hospitalization in pediatric sickle cell disease.

In a cohort of children with sickle cell disease (SCD) and vaso-occlusive pain visits served through South Carolina's Medicaid system over a 6-year period (N 5 523), we compared the number of vaso-occlusive pain or acute chest syndrome (ACS)/pneumonia episodes, and outpatient or acute service costs in those treated or not treated with hydroxyurea (HU). HU may be an underused intervention for SCD in this practice setting, for a variety of reasons. Treatment with HU varied greatly, appears to have been administered to more severely ill children, but was associated with a reduction in vaso-occlusive pain episodes, hospitalizations,and total costs of care within the HU cohort during a 2-3 year period of active HU treatment. Those receiving care through specialized SCD clinics were less likely to have pain or acute care episodes(RR 5 0.79, P < 0.0001; RR 5 0.90, P 5 0.01). Compared with the non-HU cohort, the HU group evinced a significantly higher risk of experiencing vaso-occlusive pain episodes (RR 5 3.32, P < 0.0001)and ACS/pneumonia episodes (RR 5 2.66, P < 0.0001), and higher outpatient,inpatient/emergency, and total service costs (RR 5 1.85, 2.11,2.10, and P < 0.0001, respectively) over time. HU is clinically effective in reducing pain episodes, hospitalizations, and total care costs, but those receiving it might be more severely ill.

Tissue plasminogen activator for occluded peritoneal dialysis catheter.

Inadequate flow in a peritoneal catheter is a common problem in pediatric patients. We report the effectiveness of the intra-catheter application of tissue plasminogen activator for the resolution of a malfunctioning peritoneal dialysis catheter in a child.