scholarly journals The Cost of Healthcare for Pediatric Patients with Sickle Cell Disease

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 1030-1030
Author(s):  
Maite E. Houwing ◽  
Frederick Thielen ◽  
Anne P.J. de Pagter ◽  
Jan A Hazelzet ◽  
Hedwig Blommestein ◽  
...  
Keyword(s):  
Resource Use ◽  
Research Funding ◽  
Inpatient Care ◽  
Pediatric Patients ◽  
Standard Treatment ◽  
Medical Center ◽  
Cost Of Care ◽  
Comprehensive Care ◽  
Age Group ◽  
Cell Disease

Introduction Sickle cell disease (SCD) is an autosomally, recessive inherited hemoglobinopathy and multisystem disorder characterized by ongoing hemolytic anemia, episodes of vaso-occlusion and progressive organ failure with ultimately a shortened life expectancy. Despite intensive comprehensive care and improved rates of morbidity and mortality, SCD care is still marked by high utilization of medical resources. Until now, most cost-of care studies have focused on one or two care categories, such as hospitalizations and physician visits [1-4]. Also, few studies have evaluated healthcare expenditures exclusively in children. Estimating cost-of care is important as it can ensure sufficient allocation of resources. In addition, SCD expenditures can be used to raise awareness of disease severity and serve as an incentive for prevention and management of disease complications. Primary aims of this study were to (a) investigate the overall cost of healthcare for pediatric SCD patients and to (b) estimate major cost drivers. Methods All pediatric SCD patients visiting the Erasmus University Medical Center-Sophia Children's Hospital for routine or emergency care from January 1st to December 31st 2017 with a diagnosis of SCD were included. Retrospective data of this cohort were analyzed for 24 months during January 1st 2015 to December 1st 2016. Patients were grouped into four age categories; (A) 0-12 months, (B) 1-5 years, (C) 5-13 years and (D) 13-19 years. For patients born before January 1st 2015, each individual contributed two years of follow-up time. As some children were born during one of the two years during the study time period, the weighted average was calculated based on the time patients were potentially able to make costs. Healthcare utilization of included patients was based upon data from two main sources. The clinical SCD standard treatment guideline was used to determine the expected resource use of routine comprehensive care (planned elective care) and the Erasmus University Medical Center financial claims database was used to estimate real-world resource use associated with acute and inpatient care (additional care). The included items for the SCD guideline and financial claims database per cost category are summarized in Table 1. Results A total of 125 patients were analyzed. The mean age was 8.1 years (SD: 5 years) on December 31st 2015 and 9.9 year (SD: 5 years) on December 31st 2016. Expenditures for the 125 children with SCD averaged €4285.09 (SD: €820.36) per child per year. The majority (49%) of costs was associated with standard treatment (i.e. prophylactic antibiotics); 23% with diagnostics; 19% with inpatient hospital care and 9% with outpatients visits. Annual average costs per patient per age group are depicted in Figure 1. Total expenditures for children with SCD increased per age group, ranging from €2962 (category A), €3726 (category B), and €4087 (category C) to €5890 (category D). This was mostly explained by increases in admission costs. Discussion Although healthcare utilization and costs of pediatric SCD patients have been studied previously [5-7], studies from Europe comprehensive care centers are scarce and have been mostly based on only one aspect of care such as hospitalizations costs [2]. To our knowledge, this is the first study combining standard treatment costs with real world resource use. The total annual coast of healthcare for children with SCD, including inpatient care, outpatient care, diagnostics and treatment averaged €4285.09 per patient per year. This is much lower when compared to costs of healthcare for pediatric patients with SCD reported in other studies. Kauf et al. calculated total costs of healthcare for SCD patients aged 0-9 years to be $10.704 [7]. In addition, inpatient care accounts for a relatively small part of total costs in our study. This finding has been inline with previous research where comprehensive care has been suggested as a means of reducing costs associated with SCD care [8]. A comprehensive, multidisciplinary approach is necessary to address the physical, mental and social needs of each child and their family. Comprehensive care, with effective management in the outpatient setting is able to prevent hospital admission, and is essential for delivery of high quality cost-effective care in SCD. Disclosures Cnossen: Pfizer: Other: Travel Grants, Research Funding; Bayer: Other: Travel Grants, Research Funding; Novo Nordisk: Research Funding; Nordic Pharma: Research Funding; CSL Behring: Other: Travel Grants, Research Funding; Sobi: Research Funding; Baxter: Other: Travel Grants, Research Funding; Shire: Other: Travel Grants, Research Funding; Takeda: Other: Travel Grants, Research Funding; Roche: Other: Travel Grants; NWO: Other: Governmental grants , ZonMW, Innovation fund and Nationale Wetenschapsagenda 2018.

scholarly journals Emergency Department Encounters, Hospitalizations and ED Reliance Among Medicaid Eligible Patients with Sickle Cell Disease in North Carolina

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 2113-2113
Author(s):  
Paula Tanabe ◽  
Nancy Crego ◽  
Christian Douglas ◽  
Emily Bonnabeau ◽  
Marian Earls ◽  
...  
Keyword(s):  
North Carolina ◽  
Sickle Cell Disease ◽  
Old Age ◽  
Sickle Cell ◽  
Research Funding ◽  
Claims Data ◽  
Age Group ◽  
Cell Disease ◽  
Old Patients ◽  
The Mean

Introduction: Sickle cell disease (SCD) is a complex disease for which pain is the hallmark. Pain from vaso-occlusive episodes is the number one reason for ED visits and hospital admissions. This paper reports Medicaid claims data from NC for individuals with SCD, including: 1) ED encounters and re-encounters within 7, 14 and 30 days; 2) hospitalization and re-hospitalization within 7, 14 and 30 days; and 3) ED reliance (EDR) score. Methods: We examined Medicaid claims data from for patients with a diagnosis of SCD (ICD 9 CM codes: 282.6x, ICD 10 CM codes: D57.0x, D57.1, D57.2x, D57.4x, D57.8x) in North Carolina. Data is reported for a cohort of 2,790 patients with a diagnosis of SCD, age 1 to 65+ and enrolled at least 11 months in NC Medicaid between March 1, 2016 and February 28, 2017. ED re-encounters and re-hospitalizations within 7, 14 and 30 days were identified using the time between the date of service listed on the ED or hospital claim and the next date of service in the subsequent claim. Individual ED Reliance (EDR) score was calculated as the total number of ED encounters divided by the total ambulatory visits (outpatient + ED encounters) per enrollee, (ambulatory visits reported elsewhere). Similar to Kroner et al, an EDR of >0.33 was considered a high score. Inpatient claims were identified using a category of service code indicating hospitalization. Results: The participants in the sample (n=2790) were majority female (57.92%), lived in metropolitan areas (77.63%) and had a mean age of 23.05 years old (SD=16.06). Of the 9,075 total ED encounters, 69.86% of the total sample had an ED encounter during the 12-month study period. There was a mean of 3.25 (SD=7.38) and median of 1 (IQR = 0 - 3) ED encounters per patient for the sample. Those who were 18-30 years old had the highest mean and median ED encounters per patient (4.98, SD= 9.34 and 2, IQR 1 to 5). The 31-45 year old group had the second most, with 4.82 (SD= 11.03) total ED encounters. The percentage of the sample with an ED re-encounter within 7, 14, and 30 days was also highest among the 18-30 year old group (29.17%, 33.98% and 40.89%) followed by those 31-45 years old (23.71%, 28.49%, and 34.80%), respectively. The 31-45 age group had the second most hospitalizations/patient and re-hospitalizations. The mean EDR was highest among 18-30 year old patients (0.35) and 46.48% of this age group had an EDR of 0.33 or greater. In the 31-45 year-old age group, the mean EDR was 0.28 and 35.18% had an EDR of 0.33 or greater. The overall sample had a mean of 1.30 (SD= 2.75) hospitalizations/patient. The 18-30 year old age group also had the highest mean total hospitalizations (2.08, SD= 3.72) and mean re-hospitalizations within 7 (0.16; SD=0.77), 14 (0.41; SD=1.68), and 30 (0.82; SD=2.79) days. The 31-45 age group had the second most hospitalizations/patient and re-hospitalizations (Table 1). Conclusions: Overall, increasing age coincided with increased ED and inpatient utilization, as well as with the period of transition from pediatric to adult SCD care. Furthermore, high EDR was most prevalent in the 18-30 age group. Our study further supports the need for increased focus on acute care utilization in the 18-45 year-old age group and considerations for improved care transition interventions. Disclosures Tanabe: NIH: Research Funding; AHRQ: Research Funding. Shah:Novartis: Consultancy, Research Funding, Speakers Bureau; Alexion: Speakers Bureau; GBT: Research Funding.

scholarly journals Fragmentation of Care for Young Adults with Sickle Cell Disease in California

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4667-4667
Author(s):  
Ashley Shatola ◽  
Ann M Brunson ◽  
Theresa H.M. Keegan ◽  
Ted Wun ◽  
Anjlee Mahajan
Keyword(s):  
Young Adults ◽  
Sickle Cell Disease ◽  
Inpatient Care ◽  
Private Insurance ◽  
Steering Committee ◽  
Age Group ◽  
Cell Disease ◽  
Age Cohorts ◽  
Increased Risk ◽  
Fragmentation Of Care

Background: Sickle Cell Disease (SCD) predominantly affects people of African ancestry, causing significant morbidity and mortality. In high-income countries children, who are more likely to receive comprehensive care than adults, have better health care outcomes. Young adults (YA) are in a period of transition from pediatric to adult models of care and the effect of receiving care at multiple medical facilities (fragmentation) during this time on outcomes has not been well-described. In this study we sought to examine fragmentation of care for YA SCD patients and the association of fragmentation on mortality. Methods: Using a previously published definition, longitudinal records from the California Patient Discharge, Emergency Department, and Ambulatory Surgery datasets were used to identify SCD patients from 1991-2016. SCD patients were placed into three different age cohorts: children (10-17), YA (18-25) and adults (26-33). Each patient required the full 7 year follow up time in each age category; patients could be in multiple age cohorts. Patients with no inpatient admissions were excluded. The number of inpatient admissions during each age group period was used as a measure of disease severity (<10, 10-19, 20-29, ≥30). SCD specialty care centers (SCD SC) were determined using all SCD inpatient admissions; facilities in the top 5% based on number of unique SCD patients seen were considered SCD SC. Patients were classified as always, sometimes and never seen at SCD SC facilities in each age group. We classified care fragmentation by the number of facilities an individual received inpatient care (1, 2, 3-4 or ≥5 unique facilities) during their time in each age group. Descriptive statistics were used to describe patients' characteristics by age group. Multivariable Poisson regression was used to identify risk factors associated with fragmented care. Multivariable Cox regression was used to determine the impact of fragmented care, disease severity and care at SCD SC on all-cause mortality, conditional on surviving to 26 years of age for the YA group. Results We identified a cohort of 6,977 unique SCD patients: 1,019 children, 1,122 YAs and 1,015 adults. The YA cohort consisted of predominately African Americans (91.3%) with 43.9% male and 56.1% female. Forty-two % YAs had Medi-Cal followed by 26.7% with private insurance as first reported health insurance. Forty-four % (494) had <10, 20.1% (226) had 10-19, 11.1% (125) had 20-29 and 24.7% (277) had ≥30 inpatient admissions while in the YA age period. Twenty-two % (248) YAs were seen at 1, 25.8% (290) were seen at 2, 30.2% (339) were seen at 3-4, and 21.8% (245) were seen at > 5 inpatient facilities. YAs and older adults were seen at a similar number of facilities while children were seen at less (Figure 1). In multivariable regression models among YAs, those with private insurance (vs. Medi-Cal) were at lower risk of fragmentation (Incident Rate Ratio (IRR)=0.85, CI 0.78-0.93, p=0.0002), while those without insurance were at a higher risk (IRR 1.45, CI 1.22-1.72, p<0.0001). Patients with more admissions (vs. <10) were at higher risk of fragmentation (IRR for 10-19 =1.42, CI 1.29-1.57; IRR for 20-29 1.49, CI 1.33-1.67, IRR for >30 admissions 2.13, CI 1.98-2.43 p<0.0001). Patients who were sometimes admitted to an SCD SC (IRR=2.19, CI 1.98-2.43, p<0.0001) or never (IRR=1.15, CI 1.15-1.46, p<0.0001) were at increased risk of fragmented care compared to those who were always admitted to an SCD SC. In the multivariable mortality model, YAs with increasing number of admissions, regardless of location, were at increased risk of death (10-19 HR 2.36, CI 1.13-4.91 p=0.022; 20-29 HR 4.25, CI 2.03-8.92, p=0.0001; > 30 admissions HR 7.79, CI 4.09-14.83, p<0.0001). Fragmentation of care and always or sometimes being admitted to an SCD SC were not associated with mortality. Conclusion Most young adult SCD patients (78%) received inpatient care at >1 facility. Of all age groups, children were most likely to be seen at only 1 facility, suggesting that fragmentation of care begins in early adulthood. Young adults without insurance, patients with more frequent admissions and those who did not always receive care at an SCD SC were at higher risk for fragmented care. Young adults with more frequent admissions were also at an increased risk of mortality. The effect of specialty centers and more consistent location of care on health-related outcomes for patients with SCD requires further study. Disclosures Wun: Janssen: Other: Steering committee; Pfizer: Other: Steering committee.

Costs, Persistence, and Hospitalizations Associated with the Use of Iron-Chelating Therapies in Sickle Cell Disease in Medicaid Patients

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3152-3152 ◽  
Author(s):  
Edward P Armstrong ◽  
Grant H Skrepnek ◽  
Samir K. Ballas ◽  
Peter Kwok ◽  
Susan Snodgrass ◽  
...  
Keyword(s):  
Iron Overload ◽  
Resource Use ◽  
Healthcare Costs ◽  
Research Funding ◽  
Cox Regression ◽  
Treatment Discontinuation ◽  
Cell Disease ◽  
Pharmaceutical Corporation ◽  
One Year

Abstract Abstract 3152 Objective: Patients with Sickle Cell Disease (SCD) receiving chronic blood transfusions require iron chelation therapies (ICTs) and good adherence to ICT to avoid sequellae of iron overload. This study evaluated cost, persistence, and medication adherence to ICTs based on a nationwide sample of Medicaid recipients with SCD. Methods: This retrospective cohort study evaluated healthcare claims from 2006 to 2010 captured within the Thomson Reuters 10 states Medicaid MarketScan database. Patients with ≥ 1 SCD diagnosis code (282.6x), ≥ 2 claims for ICT medications (deferoxamine (DFO), deferosirox (DFX)), continuously enrolled for ≥ 6 months prior to ICT initiation through 365 days post initiation of ICT were included. ICT utilization was obtained using NDC codes and HCPCS codes. Patients were classified into 3 groups based on their ICT use: DFO only, DFX only, and switches from DFO to DFX. Outcomes evaluated included adherence and persistence to ICT, frequency of SCD crises related hospitalizations, and overall costs. ICT adherence was evaluated using medication possession ratio (MPR) and persistence was defined as medication refill gap of ≥ 6 weeks. Multivariate linear, logistic, and Cox regression analyses were performed after controlling for demographics, number of transfusions, capitated Medicaid coverage, pre-index cost (6months prior to ICT initiation), number of hospitalizations, adherence, and risk-adjusted case-mix comorbidity. Results: A total of 484 SCD patients receiving ICTs from 2006 to 2010 within the 10 states were identified. The average age was 18.9 ±11.2 years of age, 45% (n =218) were male, and 64.7% (n=313) were African American. A total of 25.4% (n =123) patients, switched from DFO to DFX while 1.7% (n = 8) switched from DFX to DFO during one-year post-ICT initiation. Patients receiving DFX only, DFO only, or both ICTs received an average of 2.68 (±3.2), 3.54 (±3.0), 3.59 (±3.2) transfusions during the study period, respectively. Compared to pre-ICT period, the number of transfusion claims increased in all three cohorts during the study period; the mean difference was 2.7 in DFO, 3.3 in DFX, and 4.1 in the both ICT group. The Cox regression assessing long-term medication persistency indicated a 1.37 times higher likelihood of treatment discontinuation with DFO compared to DFX. Overall MPR was low in both groups; following one year of treatment, 37.4% remained on DFX compared to 15.7% on DFO (Figure 1). No differences were observed in total healthcare costs, number of hospitalizations and associated costs for SCD-related crises between the DFX only and DFO only cohorts. Meaningful differences in treatment discontinuation between the two treatment groups did not occur until 220+ days during the study period (Figure 1). Thus, a one year timeframe may be inadequate to observe complete impact of ICT adherence on sequellae of iron overload and subsequent resource use. Lengthening the study period beyond one year may be necessary to fully capture the long term impact of adherence on outcomes related to iron overload. Conclusion: This study indicated that there were more switches from DFO to DFX, higher medication persistency rate with DFX than DFO, and similar total healthcare costs, numbers of SCD crisis-related hospitalizations, and SCD crisis-related costs between DFO & DFX cohorts. Treatment discontinuation was generally high for all groups, especially for those on DFO, with a one year discontinuation rate of approximately 85%. Future research using a longer observation period than one year will be necessary to assess effects on long term clinical outcomes, resource use, and healthcare costs between different ICTs. Kaplan-Meier Graph for Treatment Discontinuation Between Deferoxamine and Deferasirox Disclosures: Armstrong: Novartis Pharmaceutical Corporation: Consultancy, Research Funding. Skrepnek:Novartis Pharmaceutical Corporation: Consultancy, Research Funding. Ballas:Novartis Pharmaceutical Corporation: Consultancy. Kwok:Novartis Pharmaceutical Corporation: Employment. Snodgrass:Novartis Pharmceutical Corporation: Employment. Sasane:Novartis Pharmaceutical Corporation: Employment.

Transition From Pediatric To Adult Care Of High Cost Patients In a Medicaid Population With Sickle Cell Disease

Blood ◽  
2013 ◽  
Vol 122 (21) ◽  
pp. 2927-2927
Author(s):  
Morey A. Blinder ◽  
Medha Sasane ◽  
Jonathan Fortier ◽  
Carole S. Paley ◽  
Mei S. Duh ◽  
...  
Keyword(s):  
Blood Transfusion ◽  
Healthcare Costs ◽  
Research Funding ◽  
Age Groups ◽  
Age Group ◽  
Cell Disease ◽  
Adult Care ◽  
Hospitalization Costs ◽  
Yearly Cost ◽  
Total Average

Abstract Introduction Though healthcare costs associated with the management of sickle cell disease (SCD) can be high, costs are not evenly distributed both across patients and across age groups; a small fraction of SCD patients incur a large share of healthcare costs and costs tend to increase as patients transition from pediatric to adult care setting. The aim of this study was to identify high cost SCD patients (HCSPs) and analyze cost patterns throughout lifetime and as they transition from pediatric to adult care. Method State Medicaid data from FL (1998-2009), NJ (1996-2009), MO (1997-2010), IA (1998-2010), and KS (2001-2009) were used for this study. Patients with ≥2 SCD diagnoses (ICD-9 282.6x) and ≥1 blood transfusion were included in the analysis. Patients were followed for as long as they were enrolled in Medicaid. HCSPs were defined as the fraction of most expensive patients accounting for 50% of the total average yearly cost. The share of the average total yearly cost represented by HCSPs was analyzed by age group and by type of setting (inpatient [IP], outpatient [OP], emergency department [ED], and other). Understanding that periodic events associated with high costs are likely to be responsible for high total costs, high cost events (HCEs), defined as quarters with costs ≥$33,095, corresponding to the amount separating the top 5% most expensive quarters observed in the sample, were analyzed. A longitudinal logistic regression model was used to assess associations between HCEs and transition age (16-20 years old), transfusions, hydroxyurea use, and SCD complications (pain, stroke, leg ulcers, avascular necrosis, infections, as well as pulmonary, renal, and cardiovascular events). Other covariates included transfusions during the previous quarter, other relevant medications (e.g.: pain medication, diuretics, anticoagulants), comorbidities (e.g.: hypertension, myocardial infarction, liver disease), and, serving as proxies for overall health status, the frequency of OP, IP, and ED visits during the previous quarter. Results From the cohort of 3,208 SCD patients (FL: 1,550, NJ: 992, MO: 489, KS: 121, IA: 56) who were eligible, 449 (14%) accounted for 50% of the total average yearly cost and were classified as HCSPs. HCSPs were observed on average (standard deviation [SD]) 6.39 (3.30) years, compared to 6.57 (3.24) years for other patients (p=0.2697). The average (SD) yearly total costs of HCSPs was statistically significantly higher at $108,524 ($52,900) per year compared to $17,683 ($15,037) per year for other patients (p<.0001). The share of the total yearly costs of HCSPs increased from 34.4% to 46.3% between age groups 11-15 and 16-20, reaching its maximum at 65.2% in the 26-30 age group. Average (SD) yearly hospitalization costs were $56,669 ($67,287) for HCSPs aged 11-15, accounting for 61.9% of their average total yearly costs. Average (SD) hospitalization costs increased sharply in transitioning HCSPs to reach $81,918 ($87,919), representing 74.9% of their average total yearly costs. Accordingly, the frequency of HCEs increased by 122.6% in the transitioning group from 0.110 HCE/year among patients aged 11-15 to 0.244 HCE/year among patients aged 16-20. The regression analysis indicated that patients were more likely to have a HCE during the post-transition period (age group 16-20, odds ratio [OR]: 1.41, p=.0046; age group >20, OR: 1.62, p=.0024) and when experiencing an SCD complication (OR: 3.79, p<.0001). Blood transfusions received during the previous quarter were associated with a lower likelihood of HCEs (OR: 0.87, p=.0080). Conclusion In this sample of Medicaid patients with SCD, 14% of patients were responsible for 50% of total yearly healthcare costs. Inpatient services accounted for the largest share of costs and this share increased in parallel with the frequency of high cost events in patients transitioning from pediatric to adult care, consistent with the increasing rate of SCD-related complications and inpatient admissions during that period reported in previous studies. Directing appropriate and targeted interventions including prophylaxis blood transfusion when indicated can help assist providers improve outcomes and lower healthcare costs in this patient population. Disclosures: Blinder: Novartis Pharmaceuticals: Consultancy, Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Fortier:Novartis Pharmaceuticals: Research Funding. Paley:Novartis Pharmaceuticals: Employment. Duh:Novartis Pharmaceuticals: Research Funding. Vekeman:Novartis Pharmaceuticals: Research Funding.

scholarly journals Benefits of Aquatic Therapy in Pediatric Patients with Sickle Cell Disease

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 4846-4846
Author(s):  
Sarah Leonard ◽  
Lindsay Cortright ◽  
Dmitry Tumin ◽  
Lora Joyner ◽  
Sruthipriya Sridhar
Keyword(s):  
Pain Management ◽  
Sickle Cell Disease ◽  
Sickle Cell ◽  
Pediatric Patients ◽  
Medical Center ◽  
Management Strategies ◽  
Cell Disease ◽  
Aquatic Therapy ◽  
Positive Effects ◽  
Pharmacologic Pain Management

Introduction: Individuals with sickle cell disease (SCD) experience both acute vaso-occlusive pain (VOC) and chronic pain, both primarily treated with opioids. However, opioids only address the sensory dimension of pain and repeated use of opioids can cause short and long-term side effects such as respiratory system depression, increased risk of cardiovascular events, and hyperalgesia. Non-pharmacologic approaches to the management of acute and chronic SCD pain, such as cognitive behavioral therapy, biofeedback, acupuncture, hypnosis, and megavitamins, have shown positive effects for pain management and may reduce opioid use. Among non-pharmacologic pain management methods, aquatic therapy has been underutilized and understudied in patients with SCD, despite showing benefits for pain management and quality of life in adults with musculoskeletal conditions. At Vidant Medical Center, aquatic therapy has been introduced for patients with SCD in 2015 and has been increasingly used since then. To describe the use of aquatic therapy and outcomes in pediatric patients with SCD, we retrospectively evaluated the use of this therapy at Vidant Medical Center among children hospitalized for acute VOC pain. Methods: The study was approved by the Institutional Review Board at East Carolina University. Children with SCD between 7-18 years of age at the time of admission for acute VOC pain between the years 2015-2018 were included in the analysis. During the review period, orders for aquatic therapy were placed at the provider's discretion. Our primary outcome was utilization of aquatic therapy during a given hospitalization, determined by retrospective query of the electronic medical record (EMR). Secondary outcomes include length of stay and time to readmission for VOC. Covariates were assessed at the time of the index admission included age, sex, and insurance coverage. Mixed-effects logistic or Poisson regression models were fitted to account for multiple hospitalizations per patient. Results: The analysis included 316 hospitalizations of 87 patients (48% female; median age at the earliest hospitalization, 11 years). The median duration of hospital admissions was 4 days (interquartile range [IQR]: 3, 7). Aquatic therapy was used in 38% of admissions, with a trend of increasing use during the study period (Figure 1). On multivariable logistic regression analysis of aquatic use during a given admission, characteristics associated with greater likelihood of aquatic therapy use included older age and more recent year of admission. Aquatic therapy was associated with 69% longer hospital stays, although this may be related to availability of aquatic therapy services. Among 52 patients with multiple admissions, use of aquatic therapy during a given admission was associated with 26% more days between hospitalizations (incidence rate ratio = 1.26; 95% CI: 1.21, 1.31; p=<0.001). Conclusion: Research into the use of non-pharmacologic pain management strategies for sickle cell disease has recently increased in order to better address the multidimensional aspects of pain. Of these management strategies, aquatic therapy has been very underutilized and under researched. Analysis of the utilization of aquatic therapy in our institution showed improved use over the last 3 years as well as increased duration between hospitalizations after the use of aquatic therapy. These positive results point towards the need for more research into other outcome measures derived from the use of aquatic therapy in pain management of pediatric SCD patients. Figure 1 Disclosures No relevant conflicts of interest to declare.

Baseline Characteristics of Patients with Sickle Cell Disease in An Ongoing 5-Year, Prospective, Noninterventional Registry Trial

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 1060-1060
Author(s):  
Matthew M. Heeney ◽  
Brigitta U. Mueller ◽  
Brad Baltz ◽  
Carole Paley ◽  
Jason Esposito ◽  
...  
Keyword(s):  
Medical History ◽  
Research Funding ◽  
Pediatric Patients ◽  
Karnofsky Performance Status ◽  
Performance Status ◽  
Relative Proportion ◽  
Current Treatment ◽  
Treatment Patterns ◽  
Cell Disease ◽  
Baseline Characteristics

Abstract Abstract 1060 Introduction: The baseline characteristics and therapeutic management of sickle cell disease (SCD) can be quite variable depending on disease severity. This report describes baseline assessments and therapies of 420 patients in an ongoing registry designed to study current treatment patterns, natural history, and outcomes in patients with SCD. Methods: This registry trial involving 57 US hematology centers had a sequential recruitment target of 600 patients with SCD subdivided into 3 age groups (2-<12 yr, 12-<18 yr, ≥18 yr). Patients '2 years of age with HbSS, HbS/β-thalassemia, or HbSC were eligible. Follow-up visits were every 6 months for a maximum of 5 years. Assessments included demographics, SCD history, Karnofsky Performance Status, medical history, types of crises, frequency of crises and hospitalization, treatment history, transfusion practices and use of iron chelation. Differences between pediatric (<18 yrs) and adult (≥18 yrs) patients were examined. (ClinicalTrials.gov identifier, NCT01220115). Results: Enrollment: 420 patients (254 <18 yrs, 166 ≥18 yrs) completed the initial baseline visit. Baseline characteristics and medical history are shown in the Table and Figure. Conclusions: These data indicate differences in baseline characteristics and treatment patterns between adult and pediatric patients with SCD in this registry. Karnofsky performance status was worse, and serum ferritin levels were higher in adult versus pediatric patients. The percentage of patients having regular transfusions was higher in pediatric patients, while adult patients had higher rates of intermittent transfusions. As expected, the lifetime chelation exposure was more frequent in adult patients. Hospitalizations and use of hydroxyurea were also greater in pediatric patients. In terms of medical history related to SCDs, pediatric patients had higher frequencies of asthma, abnormal TCDs, dactylitis and splenic sequestration, while adults had more frequent evidence of avascular necrosis, gall bladder disease, leg ulcers and pulmonary hypertension. Pain was the most common crisis type in both cohorts, although this accounted for a greater relative proportion of crises in the adult cohort. The relative proportion of ACS events was lower in the adult versus pediatric patients; all other crisis types occurred in similar relative proportions in both cohorts. This ongoing registry will provide information about disease patterns, current treatment practices, and outcomes, thus contributing to a better understanding of the appropriate therapeutic management of patients with SCD. Disclosures: Heeney: Novartis: Consultancy, Research Funding. Baltz:Novartis: Research Funding. Paley:Novartis: Employment. Esposito:Novartis: Employment. Sharma:Novartis: Employment. Vichinsky:Novartis, Apotex, Ferrokin: Research Funding.

scholarly journals Adherence to Iron Chelation Therapy and Associated Healthcare Resource Utilization and Costs in Medicaid Patients with Sickle Cell Disease

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2177-2177
Author(s):  
Francis Vekeman ◽  
Medha Sasane ◽  
Wendy Y Cheng ◽  
Agnihotram V Ramanakumar ◽  
Jonathan Fortier ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Emergency Room ◽  
Resource Utilization ◽  
Sickle Cell ◽  
Research Funding ◽  
Chelation Therapy ◽  
Iron Chelation ◽  
Cost Of Care ◽  
Iron Chelation Therapy ◽  
Cell Disease

Abstract Introduction: While adherence to iron chelation therapy (ICT) is critical for successful iron overload (IO) treatment among patients with sickle cell disease (SCD), published data indicate it is often suboptimal. Deferoxamine (DFO) and Deferasirox (DFX) are ICTs indicated for the treatment of chronic IO in patients with SCD. Lack of patient adherence may impact patient outcomes and increase cost of care. This study evaluated the economic burden of ICT non-adherence in patients with SCD from the state Medicaid program’s perspective. Methods: Patients with SCD were identified from Florida, Iowa, Kansas, Mississippi, Missouri, and New Jersey (1997-2013) state Medicaid programs. Patients were required to have ≥1 ICD-9 diagnosis code for SCD (282.6), ≥1 prescription for DFO or DFX, and ≥6 months of continuous enrollment prior to the 1st DFO/DFX prescription (index date), which was defined as the baseline period. Adherence was estimated using the medication possession ratio (MPR), defined as the sum of the days of medication supply divided by the number of days between 1st and last prescription fill plus the days of supply of the last fill; a threshold of ≥0.80 was used to define optimal adherence. All-cause and SCD-specific resource utilization per-patient-per-month (PPPM) was assessed using cumulative rates, accounting for all visits observed, and compared between adherent and non-adherent patients using cumulative rate ratios (CRR). All-cause and SCD-specific healthcare costs were computed using mean cost PPPM. Regression models adjusting for baseline characteristics were used to assess resource utilization and cost differences between adherent and non-adherent patients. Results: A total of 846 eligible patients with SCD were included with 77 in DFO-only, 686 in DFX-only), and 83 in DFO/DFX switch cohort. Mean (SD) MPR was 0.68 (0.27) for DFO-only patients and 0.75 (0.26) for DFX-only patients (p<0.05). Among all users of ICT, 409 (48.3%) were considered adherent. Adherent patients were slightly younger (19 vs. 21 years, p=0.003) than non-adherent patients. Rates of transfusions were comparable between the two groups (mean [SD] transfusions PPPM, adherent: 0.41 [0.47]; non-adherent: 0.40 [0.54], p=0.456) at baseline. The adjusted rate of all-cause IP visits PPPM was lower in adherent versus non-adherent patients (CRR=0.87 [95% CI: 0.83, 0.91]; p<0.001). The adjusted rates of all-cause outpatient (OP) visits (1.10 [1.08, 1.13], p<0.001) and ER visits (1.06 [1.01, 1.10], p=0.010) PPPM of adherent patients were higher in adherent patients than those in non-adherent patients. A similar trend was observed in SCD-specific resource utilization except for rates of ER visits, which were similar between cohorts. From cost perspective, total all-cause and SCD-specific costs were lower in adherent versus non-adherent patients primarily due to lower IP costs (Table 1). SCD-specific ER and OP costs were similar in both cohorts. All-cause pharmacy costs were higher in adherent versus non-adherent patients. Conclusion: Published studies have reported low adherence to ICT, and a similar trend was found in this study. Adherent patients were observed to have less frequent hospitalizations and lower overall and SCD-specific IP costs compared to non-adherent patients. It should be noted that the rate of OP visits was higher in the adherent patients compared to non-adherent patients suggesting that adherent patients may be more closely monitored potentially resulting in better overall patient management and fewer hospitalizations. Additional analyses are needed to explore differences between adherent and non-adherent patients. Table 1 Costs PPPM Adherent patients (N=409) [A] Non-adherent patients (N=437) [B] Adjusted cost difference[A] – [B] P -value All-cause, mean [SD] $4,766 [$4,388] $5,304 [$4,725] -$724 0.072 Inpatient $1,911 [$3,647] $2,996 [$4,439] -$947 0.016 Emergency room $27 [$87] $40 [$88] -$203 0.104 Outpatient $580 [$697] $485 [$617] $49 0.500 Pharmacy $2,248 [$1,949] $1,783 [$1,449] $432 0.004 Pharmacy without ICT $215 [$482] $274 [$544] -$50 0.192 SCD-specific, mean [SD] $2,237 [$3,679] $3,116 [$4,301] -$952 0.0160 Inpatient $1,776 [$3,546] $2,782 [$4,268] -$855 0.0160 Emergency room $18 [$63] $28 [$69] -$199 0.1200 Outpatient $443 [$658] $306 [$548] $105 0.1120 Disclosures Vekeman: Novartis Pharmaceuticals: Research Funding. Sasane:Novartis Pharmaceuticals: Employment. Cheng:Novartis Pharmaceuticals: Research Funding. Ramanakumar:Novartis Pharmaceuticals: Research Funding. Fortier:Novartis Pharmaceuticals: Research Funding. Duh:Novartis Pharmaceuticals: Research Funding. Paley:Novartis Pharma: Employment. Adams-Graves:Novartis Pharmaceuticals: Consultancy, Honoraria, Speakers Bureau.

scholarly journals Hematopoietic Cell Transplantation in Thalassemia and Sickle Cell Disease: Report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry: 2000-2017

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 168-168 ◽  
Author(s):  
Donatella Baronciani ◽  
Ariane Boumendil ◽  
Arnaud Dalissier ◽  
Javid Gaziev ◽  
Ardeshir Ghavamzadeh ◽  
...  
Keyword(s):  
Bone Marrow ◽  
Sickle Cell Disease ◽  
Cord Blood ◽  
Cell Transplantation ◽  
Marrow Transplantation ◽  
Research Funding ◽  
Pediatric Patients ◽  
Hematopoietic Cell Transplantation ◽  
Advisory Board ◽  
Cell Disease

Abstract INTRODUCTION: Allogeneic hematopoietic cell transplantation (HCT) is a diffuse curative option for transfusion dependent thalassemia (TDT) and sickle cell disease (SCD). To verify transplant activity, distribution, demography, policies and outcomes the Hemoglobinopathy Registry was established inside the European Group for Blood and Marrow Transplantation (EBMT). After a previous analysis limited to TDT for the 2000-2010 period data (BMT 2016; 51:536-41), we performed an updated report considering TDT and SCD patients transplanted in the last eighteen years (years 2000-2017). METHODS: Data on pediatric patients transplanted between Jan 1st, 2000 through Dec 31st, 2017 were extracted by the EBMT promise Hemoglobinopathy registry database. Only first transplants were considered. Data are expressed as median with range unless specifically indicated. Survival probabilities were calculated with the method of Kaplan and Meier and expressed as means and 95% confidence intervals (95%CI). Differences between survival probabilities were tested by means of the log-rank test. RESULTS: In the above-specified period 3856 consecutive pediatric patients affected by TDT (2936, 76%) or SCD (920, 24%) were transplanted in 166 HCT centers distributed in 36 countries in Europe, Asia and Africa. Median age at transplant was 7.2 years (range 0.48-17.9). 3342 (87%) transplants were performed on patients <14 years, 514 (13%) on patients aged 14-17.9 years. 2593 (67%) patients received transplant from an HLA identical sibling donor. Figure 1 reports pediatric transplant activity inside the EBMT showing an increased numbers of patients transplanted after year 2010. After a median follow up of 24 months, the 2 years overall survival (OS) and event-free survival (EFS) were 91% (95%CI 90-92) and 86% (95%CI 85-87) for the entire population. In TDT, OS and EFS were 90% (95%CI 89-92) and 84% (95%CI 82-85), respectively. In SCD, OS and EFS were 94% (95%CI 92-96) and 92% (95%CI 90-94), respectively. In both diseases no outcome difference was recorded on the basis of year of transplant (data not shown). Source of hematopoietic cells was bone marrow in 70% of TDT transplants and 81% of SCD transplants. In both diseases better results were recorded with the use of bone marrow versus peripheral blood [OS 91% versus 85% (P< 0.001); 95% versus 84% (P= 0.004) for TDT and SCD, respectively]. Similar results were recorded when EFS was analyzed. 176 patients received hematopoietic cells from single cord blood donors. Of them only 21 were from an unrelated donor. 119 patients received cord blood + bone marrow. Results of related HLA identical cord blood were similar to that of HLA identical related bone marrow (data not shown). Transplantation from an HLA identical sibling offered the best results in OS and EFS compared to other donor options (P< 0.001), both for TDT and SCD. Transplant results by donor type are reported in table 1. The threshold age for optimal transplant outcomes is confirmed 14 years, with OS 92% (95%CI 91-93), EFS 87% (95%CI 85-88) versus OS 85% (95%CI 82-89), EFS 81% (95%CI 77-85) ( P<0.001) comparing younger versus older patients in the entire populations. These differences were confirmed either in TDT and SCD. The two years incidence of cumulative extensive chronic graft versus host disease was 4.1% in TDT and 4.4 % in SCD (P=ns). CONCLUSIONS: Allogeneic HCT for TDT and SCD is a widely available curative approach; the procedure has been increasing and internationally performed during the years with excellent results. The emerging gene therapy approach will have to be compared to these well-established results. Disclosures Zecca: Chimerix: Honoraria. Forni:Novartis: Other: travel expenses, Research Funding; Celgene: Research Funding; Roche: Consultancy; Shire: Research Funding; Apopharma: Other: DSM Board. Bader:Medac: Patents & Royalties, Research Funding; Neovii: Research Funding; Novartis: Consultancy, Speakers Bureau; Riemser: Research Funding; Cellgene: Consultancy. Angelucci:Vertex Pharmaceuticals Incorporated (MA) and CRISPR CAS9 Therapeutics AG (CH): Other: Chair DMC; Novartis: Honoraria, Other: Chair Steering Comiittee TELESTO Protocol; Roche Italy: Other: Local (national) advisory board; Jazz Pharmaceuticals Italy: Other: Local ( national) advisory board; Celgene: Honoraria, Other: Chair DMC.

scholarly journals Management of Severe Chronic Pain with Methadone in Pediatric Patients with Sickle Cell Disease

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 3663-3663
Author(s):  
Zachary LeBlanc ◽  
Chris Vance ◽  
Jie Zhang ◽  
Sabrina R Macksoud ◽  
Steffane Battle ◽  
...  
Keyword(s):  
Chronic Pain ◽  
Research Funding ◽  
Pediatric Patients ◽  
Baseline Period ◽  
Chronic Pain Management ◽  
Cell Disease ◽  
Severe Chronic Pain ◽  
Ed Visits ◽  
The Mean

Abstract Introduction Patients with sickle cell disease (SCD) experience acute painful events; some patients develop chronic pain requiring daily short acting opioids, and frequent emergency department (ED) visits, and inpatient hospitalization. From the Cooperative Study of Sickle Disease (Platt, NEJM 1991), only 1% of patients with SCD experience more than 6 pain events per year. Methadone is a synthetic opioid used in chronic pain, but there is a paucity of data on its use and effectiveness in the pediatric SCD population. We hypothesized that methadone can be used safely in pediatric patients with SCD with severe chronic pain and would reduce ED visits and hospitalizations. Methods We conducted a retrospective cohort study (IRB approved) of 16 pediatric patients with SCD who received methadone for chronic pain management, indicating having more than 5 pain events per year. These patients were among 1100 total patients with SCD at our center. Primary SCD providers escalated doses to maximal effect and when clinically stable, doses were weaned. Follow-up started at time of methadone initiation and ended when a patient was weaned or transitioned to adult care; the 1 year period before treatment initiation was defined as the baseline period. We calculated descriptive statistics to characterize the study population. We compared clinical outcomes (ED visits or hospitalization for pain) for the baseline period and 1 year post methadone initiation using paired T test. To evaluate methadone dosing and dependency, we identified the mean dose, percentage of time at highest dose, and wean of methadone. We assessed withdrawal and safety by reviewing outpatient encounters, EKG screening for QTc > 450, eGFR by cystatin C to determine either a decrease in eGFR by 20% or an eGFR <80, and microalbumin/creatinine urinary excretion. Results Among the 16 participants, 10 (62.5%) were male and the genotypes of each patient were: 14 SS, 1 S/β0-thalassemia, 1 SC. The mean age was 15.5 (±2.8) years at time of methadone initiation and there was a medium follow-up of 2.1 years. Clinically, 13 (81.3%) had previous cholecystectomy, 6 (37.5%) had previous splenectomy, 7 (43.8%) had radiographic evidence of infarction of a long bone within the year prior to initiation of methadone, 10 (62.5%) were receiving chronic transfusion during methadone, and 10 (62.5%) received psychosocial counseling for depression and/or anxiety. The average number of ED visits per month was 0.31 (±0.27) in the baseline period and were 0.28 (±0.28, p=0.658) and 0.31 (±0.25, p=0.921) in the 1 year post and entire follow-up periods. The rate of hospitalizations per month decreased from 0.35 (±0.19) in the baseline period to 0.19 (±0.17, p=0.016) and 0.22 (±0.21, p=0.05) in the 1 year post and entire follow-up periods. The average initial starting dose was 12.5 mg and the mean highest dose was 26.6 mg. In 14 (87.5%) patients the final dose was less than the highest dose and 4 (25.0%) patients were weaned completely off of methadone. For the entire cohort, each patient spent a median of 183 days at their highest dose, a mean of 34% of the total time treated with methadone. No patients experienced signs consistent with opioid withdrawal during the follow-up period. The most common subjective complaints were sedation (12%), nausea and/or vomiting (24%) and constipation (6%). We found no documented new abnormal QTc intervals, changes in eGFR, or new onset proteinuria during the follow up period. Conclusion Methadone use in pediatric patients with SCD is well-tolerated and may lead to improved pain control as evidence by significant reduction in hospitalizations. Additionally, when followed closely, methadone can be used safely without concern for physiologic dependency; this is supported by our ability to wean patients off methadone entirely or weaning to a lower dose than their highest dose in the majority of the cohort. Hematologists should consider methadone as a part of chronic pain management in patients with SCD and severe chronic pain. Disclosures Lebensburger: ASH: Research Funding; NHLBI K23: Research Funding.

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