Review of economic studies of sickle cell disease and adherence to hydroxyurea and clinical and economic outcomes among Texas Medicaid enrollees with sickle cell disease

Sickle cell disease (SCD) is a rare, inherited blood disorder that affects approximately 100,000 Americans. SCD was recognized as a societal health burden due to pain crises and complications resulting in costly healthcare utilization. This dissertation: 1) summarized the economic burden of SCD in the U.S.; 2) assessed hydroxyurea medication adherence and its association with vaso-occlusive crisis (VOC)-related events and healthcare utilization and costs. The systematic review (N=21 articles) revealed mean total SCD healthcare costs (2019 USD) ranged from $19,464 to$32,446/patient/year, which was 1.7 to 2.9 times greater than the overall population. The retrospective cohort study used Texas Medicaid data from 09/01/2011-08/31/16 with individuals who: 1) were between 2-63 years, 2) had at least one hydroxyurea prescription claim and were treatment naive, and 3) were continuously enrolled. Medical and pharmacy claims were analyzed for over one year to examine their medication use patterns (hydroxyurea adherence and persistence, and pain medication use) and VOC-related healthcare utilization and costs. The association between hydroxyurea adherence (Medication Possession Ratio; MPR) and VOC-related healthcare utilization were assessed using multivariate analyses. A total of 1,035 patients (18.8±12.5 years) were included. Among them, 20.9% were adherent to hydroxyurea (defined as MPR≥0.8) and mean MPR was 0.50 (± 0.30). In the 12 months following hydroxyurea initiation, 771 (74.5%) of the patients experienced ≥1 VOC events (4.1±5.1). After adjusting for covariates, being adherent to hydroxyurea was significantly (p<0.05) associated with beneficial VOC-related health outcomes including a decreased risk and lower hazard rate of VOC events, fewer VOC events, and a shorter hospital LOS. Regarding all-cause and SCD-related healthcare utilization and expenditures, being adherent to hydroxyurea was significantly (p<0.05) associated with decreased hospitalizations and ED visits, increased outpatient visits and prescriptions, and lower total SCD-related healthcare expenditures. These study findings, the high economic burden of SCD and low hydroxyurea adherence, highlight the need for multi-stakeholder efforts to improve hydroxyurea adherence among the SCD population to improve both clinical and economic health outcomes.Pharmaceutical Science

The association between hydroxyurea adherence and opioid utilization among Texas Medicaid enrollees with sickle cell disease

© 2020 Academy of Managed Care Pharmacy (AMCP). All rights reserved. BACKGROUND: Individuals with sickle cell disease (SCD) suffer from recurrent catastrophic pain crises that are often managed by opioid analgesics. Being adherent to hydroxyurea has been associated with decreased health care resource use for pain; however, evidence of its association with opioid use is limited. OBJECTIVE: To determine if adherence to hydroxyurea is associated with opioid use among patients with SCD. METHODS: This retrospective study used Texas Medicaid data from September 1, 2011, to August 31, 2016 (study period). The index date was the date of hydroxyurea initiation. Patients who were aged 2-63 years at the index date, had ≥1 inpatient or ≥2 outpatient SCD diagnoses during the study period, had ≥1 hydroxyurea prescription during the identification period (September 1, 2011-August 31, 2015), had no diagnosis of other indications for hydroxyurea during the study period, and were continuously enrolled for at least 12 months after the index date were included. Hydroxyurea adherence was measured using medication possession ratio (MPR). The study outcomes (measured 1-year post-index) were (a) opioid use; (b) number of opioid prescriptions; (c) strong opioid use (morphine, hydromorphone, fentanyl, and methadone); (d) number of strong opioid prescriptions; (e) high-dose opioid use (≥50 mg morphine milligram equivalent [MME]); and (f) days supply for opioid prescriptions. Covariates included demographic (age and gender) and clinical (vaso-occlusive crisis [VOC], avascular necrosis, iron overload, acute chest syndrome, and blood transfusion) characteristics. Descriptive, bivariate (chi-square and Wilcoxon-Mann-Whitney tests), multiple logistic regression, and negative binomial regression analyses were performed. RESULTS: 1,146 patients (18.3 [12.3] years) met the inclusion criteria. Of these, 19.6% were adherent to hydroxyurea (defined as MPR ≥ 80%) and mean (SD) MPR was 48.3% (29.7%). In the 1 year following hydroxyurea initiation, 923 (80.5%) patients had ≥1 opioid prescription with 7.6 (9.4) opioid prescriptions per patient, while 259 (22.6%) patients had ≥1 strong opioid prescription with 1.5 (4.4) strong opioid prescriptions per patient. Average (SD) opioid dose was 41.7 (74.3) mg MME, and 27.1% had high daily MME doses (≥50 mg MME). Average (SD) opioid days supply was 83.1 (112.2) days. After adjusting for covariates, compared with being nonadherent, being adherent to hydroxyurea was associated with a 50.5% decreased risk of having strong opioids (OR=0.495, 95% CI=0.278-0.879, P=0.0165). Additionally, SCD-related complications (VOC, avascular necrosis, and iron overload) and older age were significant factors associated with opioid use and higher MME. Post hoc analyses showed that being adherent to hydroxyurea was significantly associated with lower probabilities of experiencing SCD-related complications. CONCLUSIONS: Results showed that patients with SCD are moderately adherent to hydroxyurea. Being adherent to hydroxyurea was found to be associated with a lower risk of receiving a prescription for strong opioids. Findings suggest that close monitoring and interventions to improve adherence may help mitigate strong opioid use among these patients

Association between vaso-occlusive crises and opioid prescriptions among patients with sickle cell disease: A retrospective claims-based study

© This is an open-access article distributed under the terms of the Creative Commons Attribution 4.0 International License (CCBY-4.0). View this license\u27s legal deed at http://creativecommons.org/licenses/by/4.0 and legal code at http://creativecommons.org/licenses/by/4.0/legalcode for more information. Background/Objectives: Among sickle cell disease (SCD) patients, vaso-occlusive crises (VOCs) are recurrent and unpredictable attacks of acute pain. These pain crises are often treated with analgesics, including opioids, which have been associated with misuse and overdose. The aim of this study was to examine the association between VOC events and opioid use and assess the association between opioid prescriptions and health care resource utilization among SCD patients. Methods: This was a retrospective cohort study using Texas Medicaid medical and prescription claims between September 2011 and August 2016. The index date was the first SCD diagnosis. Patients (2-63 years) with at least one inpatient or two outpatient SCD diagnoses, who were continuously enrolled during 12 months postindex, were included in the study. The primary outcome was number of opioid prescriptions, while the independent variable was number of VOC events. Covariates included age, gender, nonopioid medication use, nonstudy SCD-related medication (penicillin and folic acid) use, evidence of blood transfusions, number of SCD-related complications, number of SCD-related comorbid conditions, and Charlson Comorbidity Index score. Negative binomial regression analysis was used to address study objectives. Results: Of 3368 included patients, 1978 (58.7%) had at least one opioid prescription with a mean of 4.2 (SD=7.2). Overall, 2071 (61.5%) had at least one VOC event with an average of 2.9 (SD=4.4). The results from the negative binomial regression showed that for every increase in VOC events, the number of opioid prescriptions increased by 9.5% (Incidence rate ratio=1.095, 95% CI: 1.078-1.111; P ≤ 0.0001). Other significant covariates associated with higher opioid use included age (13 and older compared to 2-12) and increase in the number of nonopioid pain medications, nonstudy SCD-related medications, and SCD-related complications. Conclusions: The majority of SCD patients had at least one VOC event and were prescribed opioids during the 12-month study period. We found that each VOC event was associated with a 9.5% increase in the use of opioids. SCD guidelines recommend opioids for the treatment of VOC-related pain. Payers and providers should be aware of opioid use in this population, consider appropriate VOC prevention measures, and provide SCD patients with access to appropriate pain management

Fetal fibronectin testing and pregnancy outcomes among Texas Medicaid patients at risk for preterm birth

OBJECTIVES: Fetal fibronectin (fFN) testing between the 24th and 34th weeks of pregnancy in patients with symptomatic preterm labor (PTL) helps assess the risk of spontaneous preterm birth (sPTB), yet the extent of its use is unknown. We assessed use of fFN testing among Texas Medicaid enrollees with symptomatic PTL and evaluated time to infant delivery and healthcare utilization/costs. STUDY DESIGN: Retrospective cohort study using medical and pharmacy claims for Texas Medicaid enrollees. METHODS: We identified pregnant women triaged through the emergency department (ED) and hospital labor-and-delivery units with symptomatic PTL between January 1, 2012, and May 31, 2015. Patients with fFN testing prior to delivery were propensity score matched 1:1 to patients without fFN testing. Primary outcomes included time to delivery from initial PTL encounter and all-cause maternal healthcare utilization and costs. RESULTS: A total of 29,553 women met the criteria for analysis, of whom 14% had a record of receiving fFN testing. Each matched cohort included 4098 patients. Compared with those who did not, patients who underwent fFN testing had significantly more clinical risk factors (mean [SD]: 1.7 [1.1] vs 1.1 [1.0]; P \u3c.0001) and were less likely to deliver during the initial hospital stay (odds ratio [OR], 0.539; 95% CI, 0.489-0.594), deliver ≤3 days following the hospital/ED encounter (OR, 0.499; 95% CI, 0.452-0.551); and receive their first PTL diagnosis during the initial hospital/ED encounter (OR, 0.598; 95% CI, 0.539-0.665). Patients who had an fFN test, compared with those who did not, had 17.5% higher total costs (P \u3c.0001) during the 5 months prior to delivery, but had gestation lengths 9.4 days longer (24.6 vs 15.2 days) than those without testing. CONCLUSIONS: Frequency of fFN testing was low in Texas Medicaid enrollees with symptomatic PTL. Patients with fFN testing had longer gestation periods and were less likely to deliver within ≤3 days of a hospital/ED encounter for PTL. These results support the role of fFN in screening for risk for sPTB among women with symptomatic PTL