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Introduction: Patients with triple-class exposed relapsed/refractory multiple myeloma (RRMM) have poor outcomes with substantial healthcare costs. Idecabtagene vicleucel (ide-cel), a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell therapy, showed deep, durable responses in patients with RRMM in the pivotal phase 2 KarMMa trial (NCT03361748). Healthcare resource utilization (HCRU) and costs were estimated for ide-cel-treated patients in the KarMMa trial., Methods: Post-infusion costs were estimated based on HCRU data, including facility care, diagnostics, medications, and procedures. Length of stay (LOS) was extracted for inpatient and intensive care unit (ICU) care. All patients had a 14-day post-infusion inpatient stay per trial protocol. Analyses were conducted for patients treated in the United States (US), who received the ide-cel target dose of 450 × 10 6 CAR + T cells and assuming a 7-day inpatient stay., Results: Overall, 128 patients received ide-cel and were included in this analysis. Mean age was 60 years, 59% were men, and 81% were white. Mean total LOS was 23.9 days. Total estimated costs over 24 months post-infusion were US$115,614 per patient, driven by facility costs (75%; $86,385). Most costs were incurred in the first month (58%; $67,259). The scenario analysis assuming a 7-day inpatient stay showed estimated 24-month costs of $92,294. For the 54 (42%) patients who received ide-cel high dose, total costs over 24 months were $113,298 per patient., Conclusions: Extrapolation of costs based on HCRU data from patients receiving single-infusion of ide-cel in the KarMMa trial showed substantially reduced HCRU and costs over 2 years after initial treatment. Most costs were incurred during the first month after ide-cel infusion, likely attributable to the 14-day inpatient stay required by the trial protocol. These findings suggest a nominal, incremental monthly cost of care immediately after treatment, which may be lower in routine clinical practice., (© 2023. The Author(s).)

BACKGROUND: Cancer diagnostic pathways are highly variable and not clearly established in the United States, which can lead to a diagnosis process that takes more time and exposes patients to invasive or unnecessary procedures, delays in treatment, worsening patient outcomes, and elevated health care resource utilization (HRU) and health care system costs. OBJECTIVE: To investigate current trends in time to diagnosis and diagnostic-related HRU preceding the patient's cancer diagnosis across all cancer types in the United States. METHODS: A retrospective claims analysis was conducted on patients newly diagnosed with cancer identified from 2018-2019 using Optum's de-identified Clinformatics Data Mart database, which includes Medicare Advantage and commercially insured members. Patients were identified using International Classification of Diseases, Tenth Revision codes and were required to have at least 2 outpatient visits at least 30 days apart or at least 1 inpatient cancer visit without prior cancer claims. The first diagnostic test was identified based on an algorithm of a 60-day gap between diagnostic tests prior to diagnosis. The index date was defined as the first diagnostic test date or an office visit less than 4 weeks prior to the first diagnostic test date. Patient characteristics, time to diagnosis, and HRU were descriptively analyzed for all patients and by cancer type. RESULTS: Among the 458,818 patients newly diagnosed with cancer included in this analysis, the mean age was 70.6 years, approximately half were female, and most were White people (65.0%) with Medicare Advantage coverage (74.0%). Patients with cancer had an overall mean (SD) time to diagnosis of 156.2 (164.9) days and 15.4% of patients waited longer than 180 days before a cancer diagnosis. High heterogeneity among cancer types was observed, with a mean time to diagnosis ranging from 121.6 days (bladder cancer) to 229.0 days (multiple myeloma). Imaging resource use during the diagnostic pathway was high for radiology (60.7%), computerized tomography (50.8%), magnetic resonance imaging (48.6%), and ultrasound (42.6%). A total of 69.3% of patients had endoscopy without biopsy, 36.5% had endoscopy with biopsy, 62.5% had other biopsies, and most patients did general urine and serum tests (91.3%) and nongenetic cancer-specific laboratory tests (84.3%). Resource use was highly varied by cancer type but tended to increase with a longer time to diagnosis. CONCLUSIONS: The proportion of patients experiencing a diagnostic process of longer than 180 days is clinically and economically meaningful. Diagnostic-related HRU was significant and highly variable, highlighting the inefficiencies in the cancer diagnostic process in the United States and the need for policies, guidelines, or medical interventions to streamline cancer diagnostic pathways to optimize patient outcomes and reduce health care system burden. DISCLOSURES: Dr Cong is an employee of Grail, LLC, which supported this study. Drs Gitlin and McGarvey are employees of BluePath Solutions, and Ms Shivaprakash was an employee of BluePath Solutions, which received financial support from Grail, LLC, for study-related research activities. This study was sponsored by Grail, LLC, a subsidiary of Illumina Inc. currently held separate from Illumina Inc. under the terms of the Interim Measures Order of the European Commission dated October 29, 2021. The sponsor had no role in the collection, management, and analysis of the data. The sponsor contributed to study design and data interpretation.

Hematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA) is a serious complication of the transplantation process that has been consistently associated with substantially greater morbidity and mortality compared with HSCT recipients who do not develop TMA. This study aimed to systematically review published signs and symptoms of HSCT-TMA and compare patients with HSCT-TMA and HSCT recipients who do not develop TMA. Publications were identified using multiple search term variations for stem cell transplantation that were entered into the PubMed, Embase, and CINAHL databases. Two reviewers screened references at the abstract level before reviewing full texts against inclusion and exclusion criteria using a PICOS-T framework. Complication proportions were grouped by organ class and then by complication type. Meta-analyses were conducted using a random-effects model in RevMan 5.4. After 2338 references were screened, a total of 30 studies were included in our analyses. The majority of studies (n = 23; 14 adult, 5 pediatric, 4 both) examined allogeneic transplantations only. Four studies examined autologous transplantation only (all pediatric), and 3 studies included both transplantation types (all pediatric). HSCT-TMA was associated with renal dysfunction (odds ratio [OR], 11.04 for adult, allogeneic and 7.35 for pediatric, all transplantations), renal failure (OR, 2.41 for adult and pediatric, allogeneic), renal replacement therapy (OR, 6.99 for pediatric, all transplantations and 60.85 for adult, allogeneic), and hypertension (OR, 5.44 for adult, allogeneic). HSCT-TMA was associated with respiratory failure (OR, 8.00 for adult and pediatric, allogeneic), pulmonary hypertension (OR, 9.86 for adult and pediatric, allogeneic), need for pleurocentesis (OR, 5.45 for pediatric, all transplantations), noninvasive ventilation (OR, 6.15 for pediatric, all transplantations), and invasive mechanical ventilation (OR, 5.18 for pediatric, all transplantations). Additionally, HSCT-TMA was associated with neurologic symptoms (OR, 2.28 for adult and pediatric, allogeneic), pericardial effusion (OR, 2.56 for adult and pediatric, allogeneic and 8.76 for pediatric, all transplantations), liver injury (OR, 3.87 for adult, allogeneic), infection (OR, 9.25 for adult, allogeneic; 2.06 for pediatric, all transplantations), gastrointestinal (GI) bleeding (OR, 7.78 for adult and pediatric, allogeneic), and acute graft-versus-host disease grade III-IV (OR, 3.29 for adult and pediatric, allogeneic). This study represents the first systematic review of HSCT-TMA signs and symptoms. Current diagnostic criteria systems involve laboratory markers for multiorgan dysfunction, including renal dysfunction, liver injury, and general tissue damage. Diagnostic criteria include neurologic symptoms, increased need for transfusions, and hypertension. This study identified additional associations with HSCT-TMA, including increased pulmonary hypertension, respiratory failure, fever, GI bleeding, and pericardial effusion. These symptoms might be included for evaluation in future diagnostic criteria and current practice., (Copyright © 2023 The American Society for Transplantation and Cellular Therapy. Published by Elsevier Inc. All rights reserved.)

Aims: Out-of-pocket (OOP) costs may constitute a substantial financial burden to patients diagnosed with cancer. Earlier stage diagnosis and treatment of cancers may promote decreased morbidity and mortality, subsequently also lowering costs. To better understand costs experienced by patients with cancer, OOP costs by stage post-diagnosis were estimated., Materials and Methods: A retrospective analysis was conducted using Optum's de-identified Integrated Claims-Clinical dataset with Enriched Oncology, which includes data from commercially insured members (June 1, 2015-July 31, 2020). Mean annual and cumulative OOP costs (co-pay + co-insurance + deductible) (2020 USD) were reported through a 3-year period post-cancer diagnosis among adult commercially insured members (not including Medicare Advantage members) diagnosed with staged breast, cervical, colorectal, lung, ovarian, or prostate cancer between January 1, 2016 and June 30, 2020 with continuous enrollment for ≥1-month post-diagnosis., Results: A total of 7,494 eligible members were identified who were diagnosed with breast, cervical, colorectal, lung, ovarian, or prostate cancer. A greater proportion of OOP costs were incurred in year 1 post-diagnosis but remained relatively high through year 3 post-diagnosis. Cumulative mean OOP costs were as high as $35,243 (lung stage IV) per commercially insured patient by year 3 post-diagnosis and were generally higher among those diagnosed at later stages (III/IV) than those diagnosed at earlier stages (I/II) across all cancers., Limitations: Generalizability of these results is limited to those with commercial health insurance coverage. Additionally, cancer staging was dependent on accuracy of staging as recorded in the electronic medical record and as determined by Optum's proprietary algorithm using natural language processing., Conclusion: Cumulative mean OOP costs among commercially insured patients during the 3-year period post-cancer diagnosis were substantial and generally higher among those with later stage cancer diagnoses. Diagnosis of cancer at earlier stages may allow for more timely treatment and lessen patient OOP costs.

This study aimed to comprehensively assess breast, colorectal, cervical, lung, and prostate cancer screening rates and trends in the United States over time among individuals for whom screening is recommended by the United States Preventive Services Task Force (USPSTF). This retrospective study was conducted in two-year intervals from January 1, 2008 to February 29, 2020, using Optum's de-identified Clinformatics® Data Mart Database, which includes Medicare Advantage and commercially insured members. Screening-eligible individuals, who had not previously had the cancer being screened and met USPSTF criteria for screening, were identified at various time points within the study timeframe for relevant screening tests within five cancer types: breast, colorectal, cervical, lung, and prostate. In the 2020 analysis period, patients who were eligible for cancer screening included: breast: 1,620,588; colorectal: 2,763,736; cervical: 1,371,506; lung: 1,491,594; prostate: 1,126,249. Breast and cervical cancer screening prevalence rates were highest (64.4% and 63.8%, respectively), followed by colorectal (29.5%), prostate (11.7%), and lung (3.8%). Black/African American individuals and Hispanics had moderately low screening rates for cervical (58.6%) and breast (61.8%) cancer, respectively; Hispanics had the lowest screening rates for prostate cancer (6.1%). Those residing in the West had lower screening rates for breast (58.9%), cervical (62.1%), and prostate (5.6%) cancer. Screening rates remained stable over time for breast, colorectal, and lung cancer, and changed significantly for cervical (-9.5%, 2012-2020) and prostate (+7.3%, 2008-2020) cancer. Real-world cancer screening rates remain suboptimal and low, and efforts to increase screening uptake and reduce cancer health disparities remain critical., Competing Interests: The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper., (© 2022 The Authors.)

Background: Cancer represents a significant source of disease burden in the United States (US), both clinically and economically. Diagnosis and treatment of cancer at earlier stages may reduce this burden. To better understand potential impacts of earlier diagnosis, healthcare costs among patients with cancer were assessed by cancer type and stage at diagnosis., Methods: A retrospective analysis was conducted using Optum's de-identified Integrated Claims-Clinical data set with Enriched Oncology, which includes data from Medicare Advantage and commercially insured members. Adult members newly diagnosed with solid tumor cancers, cancer stage at diagnosis (diagnosed 1/1/2016-6/30/2020), and continuous enrollment for at least one month post diagnosis were identified. Patients with breast, cervical, colorectal, lung, ovarian, or prostate cancer were reported. Mean standardized costs (2020 USD) were calculated in each month on an annual and cumulative basis through four years post-cancer diagnosis. In each month, costs were calculated for those with continuous enrollment and no death reported in the month. Mean annual cost per patient was estimated by summing month one to 12 mean costs and stratifying by stage at cancer diagnosis; annual year one to four costs were summed to determine cumulative costs., Results: Among members diagnosed 2016-2020 with breast, cervical, colorectal, lung, ovarian, or prostate cancer, 20,422 eligible members were identified. Mean costs increased by stage of diagnosis across all cancers at the annual and cumulative level through year four post diagnosis. Cumulative mean costs grew over time at a relatively similar rate across stages I to III and more dramatically in stage IV, except for cervical and lung cancer where the rate was relatively stable or slightly fluctuated across stages and ovarian cancer where stages III and IV both increased more sharply compared to stages I and II., Conclusions: Mean annual and cumulative healthcare costs through year four post cancer diagnosis were significantly higher among those diagnosed at later versus earlier cancer stages. The steeper increase in cumulative costs among those diagnosed in stage IV for many cancer types highlights the importance of earlier cancer diagnosis. Earlier cancer diagnosis may enable more efficient treatment, improve patient outcomes and reduce healthcare costs., (© 2022. The Author(s).)

This retrospective study estimated postinfusion health care resource utilization (HCRU) by site of care among 303 patients with relapsed/refractory large B-cell lymphoma who received third- or later-line treatment with lisocabtagene maraleucel (liso-cel) in the TRANSCEND NHL 001 and OUTREACH trials. Inpatients ( n  = 256) had higher rates of hospitalization versus outpatients ( n  = 47; >99% vs 62%), by definition, and higher rates of tocilizumab use for cytokine release syndrome and/or neurological events (22% vs 9%). Rates of intensive care unit admission, corticosteroid use, vasopressor use, hemodialysis, and intubation were generally low and similar between groups. Median (range) total hospital length of stay was 15 (0-88) days (inpatients) and 4 (0-77) days (outpatients). Over 6 months, estimated mean postinfusion cost of care was $89,535 (inpatients) and $36,702 (outpatients). Most costs were incurred in the first month post infusion (inpatients, $50,369 [56%]; outpatients, $19,837 [54%]). Lower overall HCRU was observed with outpatient postinfusion monitoring.

Chimeric antigen receptor (CAR) T-cell therapies have demonstrated high response rates in patients with relapsed/refractory large B-cell lymphoma (LBCL); however, these therapies are associated with 2 CAR T cell-specific potentially severe adverse events (AEs): cytokine release syndrome (CRS) and neurological events (NEs). This study estimated the management costs associated with CRS/NEs among patients with relapsed/refractory LBCL using data from the pivotal TRANSCEND NHL 001 trial of lisocabtagene maraleucel, an investigational CD19-directed defined composition CAR T-cell product with a 4-1BB costimulation domain administered at equal target doses of CD8+ and CD4+ CAR+ T cells. This retrospective analysis of patients from TRANSCEND with prospectively identified CRS and/or NE episodes examined relevant trial-observed health care resource utilization (HCRU) associated with toxicity management based on the severity of the event from the health care system perspective. Cost estimates for this analysis were taken from publicly available databases and published literature. Of 268 treated patients as of April 2019, 127 (47.4%) experienced all-grade CRS and/or NEs, which were predominantly grade ≤2 (77.2%). Median total AE management costs ranged from $1930 (grade 1 NE) to $177 343 (concurrent grade ≥3 CRS and NE). Key drivers of cost were facility expenses, including intensive care unit and other inpatient hospitalization lengths of stay. HCRU and costs were significantly greater among patients with grade ≥3 AEs (22.8%). Therefore, CAR T-cell therapies with a low incidence of severe CRS/NEs will likely reduce HCRU and costs associated with managing patients receiving CAR T-cell therapy. This clinical trial was registered at www.clinicaltrials.gov as #NCT02631044., (© 2021 by The American Society of Hematology.)

Introduction: The safety and efficacy of extended-release calcifediol (ERC) as a treatment for secondary hyperparathyroidism (SHPT) in adults with stage 3 or 4 chronic kidney disease (CKD) and vitamin D insufficiency (VDI) has been demonstrated in prospective randomized clinical trials (RCTs). ERC (Rayaldee®) was approved by the Food and Drug Administration in 2016 on the basis of these prospective RCTs. The current retrospective study assessed the postlaunch data available with respect to ERC's efficacy and safety in increasing serum 25-hydroxyvitamin D (25D) and reducing parathyroid hormone (PTH) in the indicated population., Materials and Methods: Medical records of 174 patients who met study criteria from 15 geographically representative United States nephrology clinics were reviewed for 1 year before and after initiation of ERC treatment. Enrolled subjects had ages ≥18 years, stage 3 or 4 CKD, and a history of SHPT and VDI. Key study variables included patient demographics, medication usage, and laboratory results, including serial 25D and PTH determinations., Results: The enrolled subjects had a mean age of 69.0 years, gender and racial distributions representative of the indicated population, and were balanced for CKD stage. Most (98%) received 30 mcg of ERC/day during the course of treatment (mean follow-up: 24 weeks). Baseline 25D and PTH levels averaged 20.3 ± 0.7 (standard error) ng/mL and 181 ± 7.4 pg/mL, respectively. ERC treatment raised 25D by 23.7 ± 1.6 ng/mL (p < 0.001) and decreased PTH by 34.1 ± 6.6 pg/mL (p < 0.001) with nominal changes of 0.1 mg/dL (p > 0.05) in serum calcium (Ca) and phosphorus (P) levels., Discussion/conclusion: Analysis of postlaunch data confirmed ERC's effectiveness in increasing serum 25D and reducing PTH levels without statistically significant or notable impact on serum Ca and P levels. A significant percentage of these subjects achieved 25D levels ≥30 mg/mL and PTH levels which decreased by at least 30% from baseline. Dose titration to 60 mcgs was rarely prescribed. Closer patient monitoring and appropriate dose titration may have led to a higher percentage of subjects achieving an increase in 25D levels to at least 50 ng/mL and a reduction in PTH levels of at least 30%., (© 2021 S. Karger AG, Basel.)

Background: Anemia is a common complication among patients with cancer receiving chemotherapy and can cause significant costs to health plans. Objective: The objective of this study is to estimate the annual budget impact of drug treatment associated with treating cancer patients with anemia due to the effect of concomitant myelosuppressive chemotherapy (i.e., chemotherapy-induced anemia [CIA]) with erythropoiesis stimulating agents (ESAs), either darbepoetin alfa (DA) once every 3 weeks (Q3W) or epoetin alfa (EA) once every week (QW), for a large US health plan in 2014. Methods: Using a patient database from a large US health plan in 2010 (n = 14 811 119), the potential CIA patient population was determined (1842 patients each per DA and EA). A budget impact of ESA treatment on this patient population in 2014 was calculated. The analysis assumed a minimum of 2 additional months of chemotherapy from initiation of the analysis. The 2014 Centers for Medicare and Medicaid Services (CMS) reimbursement rates used were: average sales price +12% of $3.68/mcg (DA) and $11.38/1000 IU (EA), and office-based injection cost of $25.08. Results: The estimated 2014 annual average drug costs per patient with CIA were $5520 (DA) and $5833 (EA). Annual average drug costs for administrations were estimated at $100 (DA) and $301 (EA) for 2014. Per member per year (PMPY) costs for patients with CIA were estimated at $5620 (DA) and $6134 (EA) for 2014. The annual total costs per CIA population (n=1842) were estimated at $10 352 629 (DA) and $11 298 798 (EA) for 2014. Conclusion: DA Q3W has the potential to provide cost savings over EA QW in terms of annual average drug cost per patient with CIA ($313 savings), PMPY costs for patients with CIA ($514 savings), and total cost per CIA population ($946 169 savings).

Purpose: The erythropoiesis-stimulating agents (ESAs), darbepoetin alfa (DA), and epoetin alfa (EA) differ with respect to dosing schedule in chemotherapy-induced anemia. DA can be administered less frequently than EA, which may increase synchronicity between chemotherapy and ESA schedules. This study compared DA and EA with respect to frequency of synchronization and frequencies of total and ESA healthcare visits in current clinical practice., Methods: A retrospective analysis of ESA utilization during ESA episodes of care was conducted on all cancer patients identified in the SDI health oncology electronic medical records database who underwent chemotherapy and received ESA therapy from July 1, 2007 to March 31, 2010 (n = 6522 DA, n = 3,439 EA)., Results: The frequency of synchronization (chemotherapy and ESA therapy on the same day) was higher with DA (67 %) than EA (58 %) (p < 0.001). The odds that an ESA administration was synchronized with chemotherapy were higher with DA compared with EA (odds ratio = 1.46, 95 % CI: 1.37, 1.54). Compared with EA, DA patients had 2.3 fewer visits with an ESA administration (p < 0.001) and 3.0 fewer total visits (p < 0.001)., Conclusions: Compared with patients receiving EA, DA patients were more likely to have an ESA administration on the same healthcare visit as chemotherapy and had fewer visits for any cause or for ESA administration. These results suggest that through greater synchronization of ESA and chemotherapy administrations, DA may reduce patient and practice burden and healthcare utilization.

Mothers and fathers of 29 girls with Rett syndrome provided data about their levels of parenting stress, marital adjustment, and family functioning. Their scores were compared to normative and clinical samples. The parents of girls with Rett syndrome reported more stress, lower marital satisfaction, and certain adaptations in family functioning compared to norms. However, most parents scored in the normal range on most measures and their scores were not related to SES. There was little relationship between specific characteristics of the daughter with Rett syndrome, such as her age and level of functioning, and her parents' scores on these measures. There were few significant differences between mothers' and fathers' scores. Results are discussed in terms of patterns of family adaptation and coping. Clinical implications are also discussed.