scholarly journals Accuracy of Administrative Coding for Sickle Cell Disease

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 3260-3260
Author(s):  
Michelle Ting ◽  
Shyamli Sinha ◽  
Timothy L. McCavit
Keyword(s):  
Sickle Cell Disease ◽  
Likelihood Ratio ◽  
Acute Care ◽  
Administrative Data ◽  
Predictive Value ◽  
Research Funding ◽  
Negative Likelihood Ratio ◽  
Senior Author ◽  
Cell Disease ◽  
Inpatient Hospitalization

Abstract Background Administrative data are increasingly used in sickle cell disease (SCD) research to study large numbers of patients at low cost. However, the validity of research with these data depends on the accuracy of administrative coding, which has been understudied in SCD. In particular, the validity of ICD-9CM coding for SCD's clinical hallmark, vaso-occlusive crisis (VOC), has not been reported. Therefore, we aim to describe the accuracy of ICD-9CM coding for VOC, acute chest syndrome (ACS), and acute splenic sequestration crisis (ASSC) in addition to SCD genotypes. Methods Administrative coding for all acute care visits (emergency department [ED], inpatient observation, and inpatient hospitalization) in SCD patients was captured for the 2013 calendar year at Children's Medical Center Dallas (CMCD) by query of administrative records. SCD visits were identified using the ICD-9CM codes 282.4x and 282.6x in the primary or any of 14 secondary code positions. From the administrative data, VOC was defined by the use of the "in crisis" codes (282.42, 282.62, 282.64, or 282.69). ACS was defined by 517.3 and ASSC by 289.52. Genotypes were defined as HbSS - 282.61 or 282.62; HbSC - 282.63 or 282.64; and HbS-BetaThal - 282.41 or 282.42. For the chart review, all visits were independently evaluated by two reviewers with the senior author settling disagreements. Previously-published consensus definitions were used for the complications of interest: VOC was defined as new onset of pain lasting at last 4 hours for which there is no other explanation; ACS was defined by new pulmonary infiltrate on chest X-ray with associated fever or respiratory symptoms; and ASSC was defined as splenic enlargement leading to anemia (hemoglobin >2g/dL under baseline), typically with thrombocytopenia. The primary reviewers and senior author evaluated 10 cases for the purpose of consensus-building before beginning data abstraction. Data capture included demographics and cause of the visit. The sensitivity, specificity, positive predictive value (PPV), negative predictive value (NPV), positive likelihood ratio (LR+), and negative likelihood ratio (LR-) of ICD-9CM coding for VOC, ACS, ASSC, and the SCD genotype were calculated. The LR+ and LR- were prevalence-weighted. Results From 448 patients, 1107 acute care visits were identified at CMCD in 2013. Patients had a median age of 8.2 years (range 0.1 - 33.9 years). Females accounted for 53% of visits. Inpatient hospitalization accounted for 374 visits (34%) whereas ED-only visits accounted for 681(61%) and inpatient observation 52 (5%). The prevalence of SCD genotypes included 69% HbSS, 19% HbSC, and 7% HbS-BetaThal. VOC, ACS, and ASSC were the "true cause" for 36% (n=402), 6% (n=68), and 1% (n=13) of acute care visits, respectively. The results of the primary analyses are displayed in the table. Agreement between reviewers for VOC and ACS was 91% (kappa = 0.80) and 99% (kappa = 0.92), respectively. When VOC was falsely coded (n=176), the most common true diagnoses were ACS (26%), fever (24%), chronic pain exacerbation (14%), and abdominal pain (10%). When ACS was falsely coded (n=21), VOC was the most common true diagnosis (71%). Of 25 (2.2%) patients incorrectly coded for SCD, 14 had sickle trait. Conclusions ICD-9CM coding for VOC demonstrated poor accuracy; however, coding for ACS and ASSC was remarkably sensitive and specific. Genotype coding lacked sensitivity with otherwise variable results. Unfortunately, coding for SCD in ICD-10 differs minimally from ICD-9CM. Therefore, these data provide an impetus to restructure ICD coding for SCD. Table 1. Sensitivity (95% CI) Specificity (95% CI) PPV(95% CI) NPV(95% CI) Postive Likelihood Ratio(95% CI) Negative Likelihood Ratio(95% CI) VOC 91.8(88.6-94.2) 75.0(71.6-78.2) 67.7(63.6-71.6) 94.1(91.8-95.9) 2.1(1.8-2.4) 0.06(0.04-0.08) ACS 92.6(83.0-97.3) 98.0(96.9-98.7) 75.0(64.1-83.5) 99.5(98.8-99.8) 3.0(2.0-4.4) 0.004(0.002-0.01) ASSC 92.3(62.1-99.6) 99.8(99.3-100.0) 85.7(56.2-97.5) 99.9(99.4-100.0) 6.0(1.6-22.0) 0.0009(0.0001-0.006) HbSS genotype 74.2(70.9-77.2) 67.7(62.5-72.6) 83.8(80.7-86.4) 53.8(49.0-58.6) 5.1 (4.2-6.1) 0.9(0.8-1.0) HbSC genotype 31.0(24.9-37.7) 99.6(98.8-99.9) 94.3(85.3-98.2) 85.8(83.5-87.9) 16.5(6.4-42.8) 0.16(0.14-0.19) HbS-BetaThalgenotype 57.1(45.4-68.2) 99.6(98.9-99.9) 91.7(79.1-97.3) 96.9(95.6-97.8) 11(4.3-28.2) 0.03(0.02-0.05) Disclosures McCavit: Pfizer: Research Funding; Novartis: Speakers Bureau; Novartis: Speakers Bureau; Gensavis LLC: Research Funding; Pfizer: Research Funding.

Outcome of Severe Vaso-Occlusive Crisis in Sickle Cell Disease Adults Admitted to Referral Centers in Africa and Europe. Introduction of Machine Learning Methods to Improve the Presev Score

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 14-15
Author(s):  
Yamna Ouchtar ◽  
Christian Kassasseya ◽  
Kene Sekou ◽  
Anne-Laure Pham Hung D'Alexandry D'Orengiani ◽  
Mehdi Kellaf ◽  
...  
Keyword(s):  
Machine Learning ◽  
Sickle Cell Disease ◽  
Decision Tree ◽  
Predictive Value ◽  
Research Funding ◽  
Low Risk ◽  
Cell Disease ◽  
Risk Patients ◽  
Decision Tree Method ◽  
Tree Method

Introduction: Sickle Cell Disease (SCD) is one of the most common genetic disease worldwide. The Acute Chest Syndrome (ACS) is a leading cause of death for SCD patients. The PRESEV1 study was set to produce a predictive score to assess the risk of an ACS development (Bartolucci et al., 2016). PRESEV2 was an international, multicenter prospective confirmatory study to validate the PRESEV score. This study aims at improving these predictions with the addition of a machine learning (ML) method. Patients and methods: Included patients follow PRESEV1 and PRESEV2 studies 'rules. The dataset thus contains 97 patients who developed an ACS episode (18.3%) against 434 patients who did not (81.7%). To compute the PRESEV score, we firstly used the method developed previously with the following variables as input: leukocytes, reticulocytes, hemoglobin levels and cervical spine pain. This method is based on a decision tree with fixed rules and is referred to as the decision tree method throughout this abstract. Secondly we used a ML method using a combined sampling method named SMOTEENN to balance the data and a C-Support Vector Classification (SVC) with fixed parameters to predict the score. This method produces a probability, with a threshold of 0.2, under which the patient is predicted to declare an ACS. We considered the dataset composed of PRESEV1 dataset and 80 percent of PRESEV2 with a randomly choice. The test dataset is thus composed of the remaining 20 percent of PRESEV2. This technique of random choice allowed us to use a 50-cross-validation and compute with Python an average score and a standard deviation (std). In order to allow comparison of the developed score with or without the addition of the ML method, rates were calculated by adding the weight of ACS representation in the dataset. Results: Among all parameters analyzed, the SVC method considered the following variables for calculation of the score: leukocytes, LDH, urea, reticulocytes and hemoglobin levels. A hundred and two adult patients with a severe VOC requiring hospitalization were included. Out of this pool of patients, 26 (25.5%) were predicted with a low risk of developing an ACS episode (SVC method). Sensibility and specificity were of 94.7% and 26.8%, respectfully. The negative predictive value (NPV) was of 95.8% and the positive predictive value (PPV) of 22.4%. Results are resumed in table 1. When compared to the PRESEV score (decision tree method), 44 patients out of 372 were identified with a low risk score (11.8%), Discussion and Conclusion: While the addition of a ML method did not allow the improvement of the sensibility or the NPV of the PRESEV score, it improved both the specificity and the PPV. The addition of artificial intelligence thus provides a better prediction with a higher percentage of "low-risk" patients. As highlighted in the international PRESEV study, this score could represent a useful tool for physicians in hospital settings, with limited beds. While the PRESEV score could allow a better management of "low risk" patients on one side, the identification of "high-risk" patients could also represent a serious advantage to physicians, as it could improve the feasibility of clinical trials for the prevention of this lethal complication in SCD patients. Disclosures Bartolucci: Innovhem: Other; Novartis: Research Funding; Roche: Consultancy; Bluebird: Consultancy; Emmaus: Consultancy; Bluebird: Research Funding; Addmedica: Research Funding; AGIOS: Consultancy; Fabre Foundation: Research Funding; Novartis: Consultancy; ADDMEDICA: Consultancy; HEMANEXT: Consultancy; GBT: Consultancy.

scholarly journals Assessing the Safety and Efficacy of Converting Adults with Sickle Cell Disease from Full Agonist Opioids to Buprenorphine

Blood ◽  
2018 ◽  
Vol 132 (Supplement 1) ◽  
pp. 856-856 ◽  
Author(s):  
Mandy David ◽  
Christopher Carroll ◽  
Ashley Lauriello ◽  
Benjamin Salzberg ◽  
Sophie Lanzkron
Keyword(s):  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Research Funding ◽  
Opioid Therapy ◽  
Cell Disease ◽  
Chronic Opioid Therapy ◽  
Full Agonist ◽  
Post Induction ◽  
Disease Modifying Therapy

Abstract Background: The management of pain in adults with sickle cell disease (SCD) is complex, with the intermingling of both acute vaso-occlusive events and chronic daily pain. Sixty percent of adults with SCD suffer with every day chronic pain. In patients with frequent acute visits we use aggressive disease modifying therapy to decrease the risk of VOC yet there remains a subset of patients who continue to have frequent acute visits for pain. In addition, there are patients maintained on high doses of oral opioids as outpatients who continue to have high levels of daily pain. There is little data that escalating doses of opioids is associated with benefit, yet significant data to support that higher doses are associated with harms. We consider these to be cases of opioid failure. Identifying therapies for these patients that improve quality of life is essential. Buprenorphine is a partial mu opioid receptor agonist and kappa antagonist, and has a very high affinity for the mu receptors, with an elimination half-life of 28-37 hours for the sublingual administration. The reduced risk of overdose, lower risk for misuse, diminished withdrawal symptoms, and blunting of opioid craving make it an appealing alternative to full opioid agonists in a subset of patients with SCD who continue to have significant pain or are unable to wean off of ineffective opioid therapy. The purpose of this report is to describe our experience converting patients with SCD and chronic pain from chronic opioid therapy to buprenorphine. Methods: Routine clinical care in our clinic includes offering buprenorphine transition to patients on chronic opioid therapy with numerous acute care visits despite the use of disease modifying therapy; or patients on chronic opioid therapy reporting significant ongoing pain. Patients are typically weaned to lower opioid doses (goal 90 oral morphine equivalents) prior to the planned induction with buprenorphine. Once patients are at the goal dose, they are asked to hold opioids for 12-24 hours prior to presentation so that they are in at least mild opioid withdrawal prior to their first dose of buprenorphine to avoid precipitated withdrawal. The patient is assessed with the Clinical Opiate Withdrawal Scale (COWs) and if their score is 5 or higher they are administered their first dose of sublingual buprenorphine. If COWs scores are less than 5, buprenorphine is not initiated and the subject is asked to return the following day or earlier if withdrawal symptoms begin. Patients are reassessed with the COWs and dosed with buprenorphine hourly until withdrawal has ceased. The dose needed to minimize withdrawal is considered the working total daily dose. Patients are sent home and return the next day to start their once daily dose of buprenorphine. Urine toxicology testing is done the day of planned induction. Data on acute visits 6 months prior and 6 months post induction and on complications associated with induction were pulled from the electronic health record. Results: 21 patients have been converted from full agonist opioids to buprenorphine from 3/2015-6/1/2018. The average age of the patients at the time of induction was 36.1 years (SD 9.1), 62% were female. Sixteen (76 %) of the patients had sickle cell anemia, the remainder had variant genotypes. The mean dose (in morphine equivalents) of full agonist opioid that patients were on prior to weaning of opioids was 196.8 mg (SD 222.7) and just prior to induction was 85 mg (SD 70 mg) with a range of 11.4 to 315 mg. Seventeen patients tolerated the induction without any complications, 2 patients had abdominal cramps but were successfully converted, 2 patients has adverse reactions (1 had numbness of tongue and 1 with worsening of asthma) and buprenorphine was stopped. Of the 19 patients successfully converted, two chose to stop buprenorphine and resume taking full opioid agonists. For the 13 patients with 6 months of follow-up post induction, the median number of acute care visits prior to induction was 12.5 and post was 4.0 (Figure). Conclusions: Adults with sickle cell disease on chronic full agonist opioid therapy can be safely converted to buprenorphine. Acute care utilization has dropped significantly for patients post induction. Assessment of patient reported outcomes such as quality of life and pain interference are being collected. Buprenorphine appears to be a safe and effective medication in the management of pain in adults with SCD. Figure. Figure. Disclosures Lanzkron: PCORI: Research Funding; GBT: Research Funding; Prolong: Research Funding; Pfizer: Research Funding; selexys: Research Funding; NHLBI: Research Funding; HRSA: Research Funding; Ironwood: Research Funding.

scholarly journals Implementation of a Standard Order Set at a Sickle Cell Acute Care Observation Unit

Blood ◽  
2019 ◽  
Vol 134 (Supplement_1) ◽  
pp. 3396-3396
Author(s):  
Renee Cheng ◽  
Rishi Patel ◽  
Sandra Kang ◽  
Frances Tian ◽  
Xu Zhang ◽  
...  
Keyword(s):  
Pain Management ◽  
Sickle Cell Disease ◽  
Length Of Stay ◽  
Acute Care ◽  
Sickle Cell ◽  
Research Funding ◽  
Mixed Effects ◽  
Data Sets ◽  
Cell Disease ◽  
Data Set

I NTRODUCTION: Acute painful vaso-occlusive crises (VOC) in sickle cell disease (SCD) are the leading cause of emergency department (ED) encounters and frequent hospital admissions. For patients presenting with an uncomplicated VOC, acute care observation units (ACOU) have previously been shown to reduce admission rates and length of stay. We wished to evaluate if implementing a standardized acute care order set (ACOS) at the University of Illinois Hospital Sickle Cell ACOU would decrease the time to first dose of analgesic medication, inpatient hospital stays, and subsequent admissions to the ACOU. METHODS: The ACOS includes standard orders for laboratory tests to monitor severity of sickle cell hemolysis, intravenous fluids, and analgesic medications including opioids. We conducted a retrospective analysis to evaluate if the ACOS enhanced workflow and improved the timeliness of treatment in patients experiencing a VOC. The ACOS was created in April 2017 and we compiled data from the three months before (January-March 2017) and after (May-July 2017) ACOS creation. We collected data on the time it took to administer the first opioid dose, admission rate, length of stay, number of acute care visits, ED visits, and inpatient hospitalizations in a three-month span, and demographics including variations in age, gender, and sickle cell disease genotype. Patient data was collected from a pharmacy-generated list of patients who received narcotics in the ACOU during the aforementioned time period. We analyzed the effect of the ACOS on the aforementioned variables. A mixed effects linear model was used to compare time to first dose of opioids and length of stay between data sets. A mixed effects logistic model was used for binary outcomes. Covariates of age (years), gender, and severity of sickle cell hemoglobin genotype (severe: HbSS, HbS beta0 thalassemia; mild: HbSC, HbS beta+ thalassemia) were included in the models. Statistical analyses were carried out in R version 3.4.3. DISCUSSION: The pre-ACOS data set contains 291 patient encounters for 76 patients with a median age of 37 years (interquartile range [IQR], 30-47 years), 66% female, and 71% with severe genotypes. The post-ACOS data set contained 289 patient encounters for 80 patients with a median age of 32 years (IQR, 27-45 years), 80% female, and 73% with severe genotypes. Implementation of an ACOS was associated with decreased time to pain management by 3.7 minutes (p=0.077) in patients presenting with uncomplicated VOC and with fewer repeat visits to the ACOU in the studied 3-month period [OR 0.35 (95% CI 0.13-1.00), p=0.049]. The median number of opiate doses received by patients in both data sets was 3. Using 3 as a cutoff, the implementation of the ACOS was also associated with more patients receiving >3 doses of opiates [OR=1.84 (95% CI 1.05-3.19), p = 0.033]. We demonstrate that in SCD patients experiencing VOC, a standardized ACOS was associated with a trend to reducing time to receiving pain management, with increased total opioid doses during the ACOU admission (suggesting better pain control), and subsequently with a statistically significant reduction in the number of repeat ACOU visits in the studied 3-month period. We have shown that a standardized ACOS that streamlines workflow in an ACOU may play an important role in delivering timely and quality care to patients with SCD. Disclosures Gordeuk: Pfizer: Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Global Blood Therapeutics: Consultancy, Honoraria, Research Funding; Modus Therapeutics: Consultancy, Honoraria; Ironwood: Research Funding; CSL Behring: Consultancy, Honoraria, Research Funding; Imara: Research Funding; Inctye: Research Funding; Inctye: Research Funding; Pfizer: Research Funding; Emmaus: Consultancy, Honoraria; CSL Behring: Consultancy, Honoraria, Research Funding; Ironwood: Research Funding; Novartis: Consultancy, Honoraria, Research Funding; Imara: Research Funding; Modus Therapeutics: Consultancy, Honoraria.

Acute Care Utilization Is More Common in Patients with Sickle Cell Disease Who Have Chronic Complications and Chronic Pain: A Preliminary Report from the Escaped Trial

Blood ◽  
2016 ◽  
Vol 128 (22) ◽  
pp. 2490-2490 ◽  
Author(s):  
Sophie Lanzkron ◽  
Carlton Haywood ◽  
Jane A Little ◽  
Joshua Field ◽  
Joseph Ryan Shows ◽  
...  
Keyword(s):  
Chronic Pain ◽  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Research Funding ◽  
Symptom Burden ◽  
Care Utilization ◽  
Cell Disease ◽  
Chronic Complications

Abstract Background: We undertook a large multisite observational study collecting prospective data on health care utilization of patients with sickle cell disease (SCD). No prospective examination of symptom burden has been undertaken in SCD since the Cooperative Study of Sickle Cell Disease, and none in the modern era, since the widespread adoption of hydroxyurea therapy. The ESCAPED trial aims to compare patient centered outcomes following management of acute painful vaso-occlusive (VOC) events in the emergency department or in the infusion center. Here, we examine acute care utilization patterns in the first 223 subjects who have completed at least 6 months of follow-up and test determinants of utilization. Methods: This is an ongoing, prospective cohort study that is recruiting across four sites (Baltimore, Cleveland, Milwaukee, and Baton Rouge). 500 adults with SCD who live in proximity to one of the study sites are being recruited and followed for 18 months. Data from visits for all acute, uncomplicated VOC are collected by chart review and patient interview. To ensure that acute visits are not missed, subjects are contacted on a monthly basis and where available statewide health information exchanges are queried. We tested for associations between subject characteristics upon enrollment and the number of acute visits during follow up using Poisson regression. Results: The average length of follow-up to date is 9.1 months with a range of 6.1-14.2 months for the 223 subjects who have been enrolled for at least 6 months. The mean number of acute visits per month for uncomplicated VOC by the cohort was 0.65 (SD 0.87) with a median of 0.35, minimum of 0 and maximum of 5. 43 subjects have had no acute visits. 59% of the cohort are female, the mean age is 35.6 (SD 12.1). 74.3% have sickle cell anemia, 42% are employed and 52% reported having chronic pain. In a multivariate model, factors associated with an independent decrease in likelihood of an acute visit were increasing age, a history of leg ulcers, graduating high school and being employed, while an increase in likelihood of an acute visit was associated with chronic complications (kidney disease, retinopathy, stroke, and avascular necrosis) and chronic pain. Conclusions: In this cohort, chronic complications like renal disease and AVN are associated with increased acute care visits. The association of chronic pain as an independent risk factor for acute visits, while intuitive, suggests that understanding and managing chronic pain may be central to mitigating pain and decreasing the need for acute care visits in the long term. The prevalence of chronic pain is high in this cohort, which likely well represents the contemporary US sickle cell community. The development of therapeutic strategies that address this significant complication of SCD are imperative if we are to both decrease symptom burden and the need for health care utilization in this population. Disclosures Lanzkron: NKT therapeutics: Research Funding; Prolong: Research Funding; Pfizer: Research Funding; Selexys: Research Funding; NHLBI: Research Funding; PCORI: Research Funding; GBT: Consultancy. Haywood:PCORI: Research Funding; NHLBI: Research Funding. Little:PCORI: Research Funding. Field:PCORI: Research Funding; NKT Therapeutics: Consultancy, Research Funding; Astellas Pharma: Consultancy, Research Funding. Shows:PCORI: Employment, Research Funding. Segal:PCORI funded: Research Funding. Saheed:PCORI: Research Funding. Robertson:PCORI: Research Funding. Proudford:PCORI: Research Funding. Kincaid:PCORI funded: Research Funding. Burgess:PCORI: Research Funding. Green:PCORI: Research Funding. Wang:PCORI: Research Funding. Seufert:PCORI: Research Funding. Brooks:PCORI: Research Funding. Griffin:PCORI: Research Funding. Piehet:PCORI: Research Funding. Frymark:PCORI: Research Funding. Varadhan:PCORI: Research Funding.

scholarly journals Early Evaluation of the Use of Crizanlizumab in Sickle Cell Disease: A National Alliance of Sickle Cell Centers Study

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 3113-3113
Author(s):  
Julie Kanter ◽  
Gerhard Hellemann ◽  
Alice J. Cohen ◽  
Deepa Manwani ◽  
Modupe Idowu ◽  
...  
Keyword(s):  
Sickle Cell Disease ◽  
Board Of Directors ◽  
Acute Care ◽  
Sickle Cell ◽  
Real World ◽  
Research Funding ◽  
Cell Disease ◽  
Advisory Committees ◽  
Level Data ◽  
National Alliance

Abstract Background: Vaso-occlusive crises (VOC) are the most common acute complication of sickle cell disease (SCD). Crizanlizumab, an anti-P-selectin monoclonal antibody, is an FDA-approved disease-modifying therapy (DMT) for SCD patients (pts) aged ≥16 yrs to reduce the frequency of VOC. To better understand its use and impact, the National Alliance for Sickle Cell Centers (NASCC) conducted a retrospective study of pts prescribed crizanlizumab from 11/2019-6/30/2021. NASCC is a non-profit organization formed to support SCD centers in delivering quality comprehensive care by setting and adopting specific standards and advocating for improved health outcomes in SCD. This study describes the largest real-world cohort of pts treated with crizanlizumab. Methods: This is a two-part study. Part 1 was to evaluate NASCC center crizanlizumab practice and to summarize data on insurance approval and the frequency of drug discontinuation. Part 2 includes pt level data to evaluate reasons for discontinuation and acute care utilization pre and post therapy. Acute care use includes day hospital/infusion, emergency department visits, and hospitalizations for VOC (excluding COVID-19). The index date for each pt is defined as the 1st crizanlizumab infusion date. Chart review (electronic health records) was used to identify all acute care visits 12 months pre-index and ≤12 months post index. Acute care data will be analyzed in aggregate. Evaluation of center-specific use of crizanlizumab, time to initial site level formulary approval and drug discontinuation were analyzed. Pt level data collection is ongoing to include sufficient time post index date. VOC characteristics will be summarized using medians, median differences (pre/post treatment), and 95% confidence intervals. Additional evaluation of effectiveness of crizanlizumab will include analysis based on number of doses received, pre-treatment VOC burden, concomitant hydroxyurea (HU) use and genotype. Results: Data includes pts prescribed crizanlizumab at 11 NASCC centers. Site- formulary approvals to use crizanlizumab varied from 12/2019-12/2020. As a result, the 1st pt to receive treatment at each site varied from 1/15/2020-1/20/2021. Mean time from site-level approval to first infusion was 77 days (range: 0-394). Over 50% of sites received insurance denials mainly due to "insufficient medical necessity" or "medication not covered by the prescription plan." Sites were able to successfully appeal denials for 71% of pts (Table 1). Treatment Delivery: Each site gives infusions over 30 minutes and the majority (64%) do not use pre-medication unless pts had reactions. Some sites use diphenhydramine/acetaminophen (3) or normal saline and ketorolac (1). All sites prescribe crizanlizumab to pts of all SCD genotypes. Pts Treated: 297 pts were prescribed crizanlizumab of whom 238 received ≥ 1 infusion. There was variation in number of pts/site (range 6-73, mean 21) due to time to site-level approval, insurance and pt population. Of these 238, 75 pts (32%) discontinued treatment (0-17 pts/site). Sites reported pts perceived lack of improvement or feeling their overall pain was increased, transportation issues and infusion related reactions (IRRs) characterized by pain as some of the reasons for discontinuation. Evaluation of real-world efficacy measured by changes in acute care utilization, including sub-analysis by genotype, pre-treatment VOC burden and concomitant HU use, are pending sample size dependent feasibility. Discussion: This is the first multi-center real-world analysis of crizanlizumab. Findings demonstrate some insurance barriers to therapy. The majority of pts who initiated crizanlizumab have remained on therapy; however, 1/3 of pts had lack of effect or barriers to care. Pt level data will include characteristics related to treatment failure or IRR. Improving the understanding of phenotype-specific response to novel therapies is essential in SCD. Conclusion: Post-approval therapies for rare diseases must undergo real-world evaluation to ensure study results translate to the community. NASCC uses defined criteria for multidisciplinary care for Alliance inclusion and findings reflect the use of DMT in such centers. This is the first NASCC study of DMT in SCD. Part 2 of the study will give early insights into the effectiveness of crizanlizumab; long term follow-up is needed for a full understanding of its utility in SCD. Figure 1 Figure 1. Disclosures Kanter: Fulcrum Therapeutics, Inc.: Consultancy; Novartis: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Forma: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Agios: Honoraria, Membership on an entity's Board of Directors or advisory committees; Beam: Honoraria, Membership on an entity's Board of Directors or advisory committees; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees; Graphite Bio: Consultancy; GuidePoint Global: Honoraria; Fulcrum Tx: Consultancy. Manwani: Novartis: Consultancy. Idowu: Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Research Funding; Global Blood Therapeutics: Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Forma Therapeutics, Inc.: Research Funding; Ironwood: Research Funding. Treadwell: National Alliance of Sickle Cell Centers: Other: Early Evaluation of the Use of Crizanlizumab in Sickle Cell Disease. Clay: GBT: Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria. Little: Hemex Health, Inc.: Patents & Royalties; Biochip Labs: Patents & Royalties. Desai: Global Blood Therapeutics: Honoraria, Research Funding; Novartis: Research Funding, Speakers Bureau; Pfizer: Other: Publication Fee, Research Funding; Forma: Consultancy; Foundation for Sickle Cell Research: Honoraria. Lanzkron: Shire: Research Funding; Pfizer: Current holder of individual stocks in a privately-held company; Bluebird Bio: Consultancy; Teva: Current holder of individual stocks in a privately-held company; Novo Nordisk: Consultancy; GBT: Research Funding; Imara: Research Funding; CSL Behring: Research Funding; Novartis: Research Funding.

Acute Care Visits in Sickle Cell Disease: a Population-Based Multi- State Study

Blood ◽  
2008 ◽  
Vol 112 (11) ◽  
pp. 165-165 ◽  
Author(s):  
David Brousseau ◽  
Julie A. Panepinto ◽  
Pamela Owens ◽  
Claudia Steiner
Keyword(s):  
Emergency Department ◽  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Population Based ◽  
Emergency Department Visits ◽  
Cell Disease ◽  
Principal Diagnosis ◽  
Inpatient Hospitalization ◽  
Entire Cohort

Abstract Background: The number of times a patient will seek acute care in the emergency department or require hospitalization for sickle cell related illness has not been described in a population-based manner. Twenty years ago, rates of acute care visits for 3,578 patients who were part of the Cooperative Study of Sickle Cell Disease were reported, eloquently describing patterns of acute care utilization for people followed at select centers. In that study, only 1% of patients had more than six visits per year and 5% of the population (who made three to 10 visits per year) accounted for almost one-third of all visits. The objective of this study is to describe the emergency department and hospital utilization for patients with sickle cell related conditions over a two- year period. This study will be the first to provide a complete assessment of the utilization patterns of patients with sickle cell disease, one that is multi-state, inclusive of all ages, all insurance types, and includes patients that are followed at community, academic and tertiary care centers. Methods: We conducted a retrospective cohort study using 2005 and 2006 State Emergency Department and State Inpatient Databases that include encrypted personlevel identifiers to allow linkage of record level information. The data are from the Healthcare Cost and Utilization Project (HCUP), a Federal-State-Industry partnership sponsored by the Agency for Healthcare Research and Quality. Data for all sickle cell related emergency department visits and hospitalizations within the following seven states (Arizona, California, Florida, Missouri, South Carolina, Tennessee, and New York) were extracted for each patient. To be eligible, a patient had at least one sickle cell specific visit, defined as a visit with a principal diagnosis of sickle cell crisis or a secondary diagnosis of sickle cell disease with a principal diagnosis that was sickle cell related (e.g. pneumonia, stroke, fever). All sickle cell related visits were linked by unique personal identifiers, thus clustering visits by patient and allowing population-based statewide assessments of utilization. An emergency department visit on the same day as an inpatient hospitalization was counted only as an inpatient hospitalization to avoid over counting care-seeking visits. The distribution of acute care visits for each patient (presented as numbers of emergency department visits and hospitalizations over the two-year period) was determined for the entire cohort, then stratified as child versus adult. Results: A total of 24,668 patients with sickle cell disease made 86,535 acute care visits during the two-year study period, 33,520 (38.7%) were emergency department visits and 53,015 (61.3%) were inpatient visits. Of the 24,668 patients, 8,895 (36.1%) were less than 18 years of age; 15,773 were adults. 52.8% of the entire cohort made one visit in the two year period. 1,320 (5.4%) patients had more than 12 visits over the two-year time period; 3,210 (13.0%) made 6–20 visits over two years, and accounted for 31,752 (36.7%) acute care visits. An additional 579 (2.4%) patients had more than 20 visits over two years, accumulating 18,701 (21.6%) acute care visits. Children were less likely to have more than 12 visits over the two years (1.9%) compared to adults aged 18–45 (8.1%) and were also less likely to be in the high utilization group of 6–20 visits over two years (9.9% of children compared to 15.8% of those 18–45 years old). Conclusions: A significant proportion of patients with sickle cell disease seek acute care multiple times in an emergency department setting or through hospitalization. Our population-based study demonstrates an increased proportion of high utilizers compared to previous work, especially among adult patients. Our findings likely reflect the difference in healthcare utilization in the broader community as compared to that within a cooperative study in academic settings. It suggests that some patients, adults in particular, may have limited access to urgent care in a primary care setting and would benefit from better access and more aggressive preventive care. Further work on patterns of and reasons for utilization, especially emergency department care, in this high-utilizer group, would be helpful in targeting and improving overall care for these patients.

Emergency Department Treat-and-Release Visits for Sickle Cell Disease: A sIgn of acute events to come

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 169-169
Author(s):  
David C Brousseau ◽  
Claudia A Steiner ◽  
Pamela Owens ◽  
Andrew Mosso ◽  
Julie A. Panepinto
Keyword(s):  
Emergency Department ◽  
Sickle Cell Disease ◽  
Acute Care ◽  
Sickle Cell ◽  
Cell Disease ◽  
Public Insurance ◽  
Inpatient Hospitalization ◽  
Inpatient Hospitalizations ◽  
Ed Visits ◽  
Return Visits

Abstract Abstract 169 Background: Patients with sickle cell disease have very high rates of rehospitalization, with rates as high as 40% for young adults. Many institutions have invested significant resources to utilize an inpatient hospitalization as a trigger to alter care and prevent further hospital utilization. While this focus on hospitalizations is important, there has been little attention given to return visits following treat-and-release emergency department (ED) visits. It has been shown that patients with sickle cell disease have high use of acute care resources, including the ED. Given that only half of ED visits by patients with sickle cell disease result in an inpatient stay, it may be possible to use an ED visit as a trigger for improved care rather than waiting for an inpatient hospitalization. We hypothesized that patients with sickle cell disease who were treated and released from the ED would have high rates of return for acute care utilization, both to the ED and the inpatient unit, within 14 days. We further hypothesized that young adults and those with public insurance would have the highest return for acute care utilization rates. Methods: We conducted a retrospective cohort study using 2005 and 2006 State Emergency Department Databases and State Inpatient Databases. The data are from the Healthcare Cost and Utilization Project (HCUP), a Federal-State-Industry partnership sponsored by the Agency for Healthcare Research and Quality. Data for all sickle cell-related ED visits and hospitalizations within the following eight states (AZ, CA, FL, MA, MO, SC, TN, and NY) were extracted for each patient. One-third of patients with sickle cell disease in the United States live within these states. All sickle cell related visits were linked via encrypted person-level identifiers to allow linkage of record level information, thus clustering visits by patient. Each treat-and-release ED visit served as an index visit; all subsequent ED treat-and-release visits and inpatient hospitalizations (whether through the ED or not) were tracked for periods of 7 and 14 days. ED treat-and-release visits within the seven days following a hospital discharge were excluded from being index visits. Results: A total of 12,109 patients with sickle cell disease made 39,775 ED treat-and-release visits during the two-year study period. Of the index ED treat-and-release visits, 4,162 (34.4%) children (ages 1–17 yrs) made 8,636 (21.7%) visits compared to 4,166 (34.4%) 18–30 year olds who made 17,070 (42.8%) ED treat-and-release visits. Overall, 16,731 (42.1%) of the ED treat-and-release visits had either an inpatient hospitalization or another ED treat-and-release visit within 14 days of the index ED visit; 39.7% of those return visits were inpatient hospitalizations meaning that 16.7% of ED treat-and-release visits are followed by an inpatient hospitalization within 14 days. Analyzing the 42.1% return visit rate by age and payer revealed that 49.0% of ED treat-and-release visits by 18 – 30 year old patients resulted in return visits compared to 24.7% of children and 38.6% of 46–64 year olds. 46.5% of ED treat-and-release visits by those with public insurance resulted in a return visit compared to 32.2% of visits by those with private insurance and 35.0% of those who were uninsured. As the timing of return visits might direct the intervention, we also evaluated 7 day return visits. Of the 16,731 return visits within 14 days, 12,561 (75.1%) occurred in the first 7 days; 41.1% of the 7 day return visits were inpatient hospitalizations meaning that 13% of ED treat-and-release visits were followed by an inpatient hospitalization within 7 days. Conclusions: A significant proportion of patients with sickle cell disease return for acute care following an ED treat-and-release visit, with young adults and those with public insurance having the highest rates of return visits. A high percentage of those return visits are hospitalizations. Given these findings, ED treat-and-release visits should serve as a trigger to focus enhanced outpatient comprehensive care on these patients in order to prevent a subsequent inpatient hospitalization and to ultimately improve care for patients with sickle cell disease. Disclosures: No relevant conflicts of interest to declare.

scholarly journals Circulating tumour DNA methylation in hepatocellular carcinoma diagnosis using digital droplet PCR

2021 ◽  
Vol 49 (3) ◽  
pp. 030006052199296
Author(s):  
Juan Wang ◽  
Liu Yang ◽  
Yanjun Diao ◽  
Jiayun Liu ◽  
Jinjie Li ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Dna Methylation ◽  
Likelihood Ratio ◽  
Predictive Value ◽  
Positive Likelihood Ratio ◽  
Negative Likelihood Ratio ◽  
Control Group ◽  
Control Subjects ◽  
Droplet Pcr ◽  
Healthy Control

Objective To evaluate the performance of a DNA methylation-based digital droplet polymerase chain reaction (ddPCR) assay to detect aberrant DNA methylation in cell-free DNA (cfDNA) and to determine its application in the detection of hepatocellular carcinoma (HCC). Methods The present study recruited patients with liver-related diseases and healthy control subjects. Blood samples were used for the extraction of cfDNA, which was then bisulfite converted and the extent of DNA methylation quantified using a ddPCR platform. Results A total of 97 patients with HCC, 80 healthy control subjects and 46 patients with chronic hepatitis B/C virus infection were enrolled in the study. The level of cfDNA in the HCC group was significantly higher than that in the healthy control group. For the detection of HCC, based on a cut-off value of 15.7% for the cfDNA methylation ratio, the sensitivity and specificity were 78.57% and 89.38%, respectively. The diagnostic accuracy was 85.27%, the positive predictive value was 81.91% and the negative predictive value was 87.20%. The positive likelihood ratio of 15.7% in HCC diagnosis was 7.40, while the negative likelihood ratio was 0.24. Conclusions A sensitive methylation-based assay might serve as a liquid biopsy test for diagnosing HCC.

scholarly journals Misuse of ultrasound for palpable undescended testis by primary care providers: A prospective study

2015 ◽  
Vol 9 (11-12) ◽  
pp. 387 ◽  
Author(s):  
Nathan C Wong ◽  
Rahul K Bansal ◽  
Armando J Lorenzo ◽  
Jorge DeMaria ◽  
Luis H Braga
Keyword(s):  
Primary Care ◽  
Physical Examination ◽  
Likelihood Ratio ◽  
Predictive Value ◽  
Undescended Testis ◽  
Positive Likelihood Ratio ◽  
Negative Likelihood Ratio ◽  
Primary Care Providers ◽  
Care Providers ◽  
Hospital Patients

<p><strong>Introduction:</strong> Although previous evidence has shown that ultrasound is unreliable to diagnose undescended testis, many primary care providers (PCP) continue to misuse it. We assessed the performance of ultrasound as a diagnostic tool for palpable undescended testis, as well as the diagnostic agreement between PCP and pediatric urologists.</p><p><strong>Methods:</strong> We performed a prospective observational cohort study between 2011 and 2013 for consecutive boys referred with a diagnosis of undescended testis to our tertiary pediatric hospital. Patients referred without an ultrasound and those with non-palpable testes were excluded. Data on referring diagnosis, pediatric urology examination and ultrasound reports were analyzed.</p><p><strong>Results:</strong> Our study consisted of 339 boys. Of these, patients without an ultrasound (n = 132) and those with non-palpable testes (n = 38) were excluded. In the end, there were 169 pateints in this study. Ultrasound was performed in 50% of referred boys showing 256 undescended testis. The mean age at time of referral was 45 months. When ultrasound was compared to physical examination by the pediatric urologist, agreement was only 34%. The performance of ultrasound for palpable undescended testis was: sensitivity = 100%; specificity = 16%; positive predictive value = 34%; negative predictive value = 100%; positive likelihood ratio = 1.2; and negative likelihood ratio = 0. Diagnosis of undescended testis by PCP was confirmed by physical examination in 30% of cases, with 70% re-diagnosed with normal or retractile testes.</p><p><strong>Conclusion:</strong> Ultrasound performed poorly to assess for palpable undescended testis in boys and should not be used. Although the study has important limitations, there is an increasing need for education and evidence-based guidelines for PCP in the management of undescended testis.</p>

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