Impact of the COVID-19 pandemic on utilization and cost for care of pediatric and young adult ALL.

OBJECTIVE: Acute lymphoblastic leukemia (ALL) is the most common childhood malignancy and among the most common malignancies in young adults and requires a unique pattern of healthcare utilization including an acute/emergent presentation and an intensive initial 8 months of therapy followed by two years of outpatient treatment. The COVID-19 pandemic caused massive global disruptions in healthcare use and delivery. This report aims to examine the effects of the COVID-19 pandemic on the presentation, diagnosis and continued management of childhood and young adult ALL in regard to utilization and cost of care among commercially insured individuals in the United States. RESULTS: Utilizing a commercial insurance claims database, 529 pediatric and young adult patients were identified who were diagnosed with ALL between January 2016 and March 2021. New diagnoses were evaluated by era and demographics. Utilization was measured by COVID-related era as number of inpatient and outpatient encounters, inpatient days, and cumulative cost during the initial 8 months of therapy. None of these cost or utilization factors changed significantly during or shortly after the pandemic. These findings reinforce that the necessary care for pediatric and young adult ALL was unwavering despite the massive shifts in the healthcare system caused by the COVID-19 pandemic. This provides a valuable benchmark as we further examine the factors that influence the pandemic's impact on health equity and access to care, especially in vulnerable pediatric and young adult populations. This is the first investigation of the effect of the COVID-19 pandemic on utilization and cost of care in pediatric and young adult cancer.

Healthcare cost and utilization for chimeric antigen receptor (CAR) T-cell therapy in the treatment of pediatric acute lymphoblastic leukemia: A commercial insurance claims database analysis.

BACKGROUND: B-lineage acute lymphoblastic leukemia (B-ALL) is the most common malignancy of childhood. With the introduction of novel cellular therapies, cost of care is a critical component and the financial burden experienced by patients and society requires evaluation. AIMS: This study aims to assess the utilization and cost of care for chimeric antigen receptor T-cell (CAR-T) therapy for pediatric ALL patients with commercial insurance coverage in the United States. METHODS AND RESULTS: Using de-identified commercial insurance data from the OptumLabs® Data Warehouse, a cohort of 37 patients, aged 1-25 years, with B-ALL treated with CAR-T therapy between Oct 2016 and Dec 2021 in the United States was identified. Cost was evaluated for a 90 day period encompassing CAR-T infusion and by administration and complication characteristics. Among the 37 identified B-ALL patients that received a CAR-T product infusion, 14 patients were female, median age at administration was 13 years. The median 90-day total cost was $620,500 (Mean: $589,108). Inpatient cost accounted for approximately 71% of the total cost with an average of 28 inpatient days per patient. Although inpatient cost was slightly higher in the older age group (aged 10-25 years) and in patients with a code for cytokine release syndrome (CRS), these differences were not statistically significant. CONCLUSION: This real-world cost analysis shows for the first time the encompassing cost of CAR-T therapy for pediatric B-ALL patients in the US with commercial insurance. This study provides a valuable benchmark that can be used to analyze the financial implications of CAR-T therapy for pediatric B-ALL therapy on health systems.

Utilization and Cost of Outpatient Rehabilitation Services for Pediatric Patients Treated for Acute Lymphoblastic Leukemia Using a Commercial Claims Database.

OBJECTIVE: To investigate the temporal trends and factors associated with outpatient rehabilitation utilization and costs for pediatric acute lymphoblastic leukemia (ALL). DESIGN: Deidentified administrative claims data and longitudinal health information on patients representing a mixture of ages, ethnicities, and geographic regions across the United States were accessed using Optum Labs Data Warehouse. Regression models were constructed to assess associations of outpatient rehabilitation with age, sex, race and ethnicity, year of diagnosis, and region. SETTING: Outpatient rehabilitation. PARTICIPANTS: 1000 Patients aged 1-30 years with a new diagnosis of ALL between 1993 and 2017 and continuous insurance coverage (N=1000). INTERVENTION: Not applicable. MAIN OUTCOME MEASURES: Outpatient rehabilitation service utilization and cost based on reimbursed charge codes, summarized over 36 months after cancer diagnosis. RESULTS: In 1000 patients, utilization of outpatient rehabilitation services increased from 20% in 1993-2002 to 55% in 2013-2017. In the earliest era examined, physical and/or occupational therapy was provided to 18% and increased to 54% in the latest years. Speech service utilization remained between 5%-8% across timepoints. Inflation-adjusted cost for provision of services did not change significantly across time and remained low, accounting for a median of 1.3% (Q1, Q3 0.3, 3.4) of total treatment cost in 1993-2002 and decreasing to a median 0.4% (Q1, Q3, 0.1, 1.0) in 2013-2017. Age 1 to 5 years at ALL diagnosis was associated with increased rehabilitation visit number and cost, and treatment in the Midwest was associated with increased likelihood of outpatient rehabilitation service utilization compared to other geographic regions. CONCLUSIONS: Outpatient rehabilitation services are being increasingly provided to patients with ALL at a relatively low cost per patient, yet geographic variability in care utilization is evident. These services do not add excessively to the overall cost of leukemia care and thus cost containment should not be an excuse to limit access.

Type I and Ir pleuropulmonary blastoma (PPB): A report from the International PPB/DICER1 Registry.

BACKGROUND: Pleuropulmonary blastoma (PPB) is the most common lung cancer of infancy and early childhood. Type I PPB is a purely cystic lesion that has a microscopic population of primitive small cells with or without rhabdomyoblastic features and may progress to type II or III PPB, whereas type Ir lacks primitive small cells. METHODS: Children with suspected PPB were enrolled in the International PPB/DICER1 Registry. Pathology was centrally reviewed, and follow-up was ascertained annually. RESULTS: Between 2006 and 2022, 205 children had centrally reviewed type I or Ir PPB; 39% of children with type I and 5% of children with type Ir PPB received chemotherapy. Outcomes were favorable, although 11 children (nine with type I and two with type Ir PPB) experienced progression to type II/III (n = 8) or regrowth of type I PPB at the surgical site (n = 3), none of whom received chemotherapy before progression. Age and cyst size in combination were more suitable than either factor alone in predicting whether a particular lesion was type I or Ir PPB. CONCLUSIONS: For young children with type I PPB, outcomes are favorable, but complete resection is indicated because of the risk for progression. Chemotherapy may be useful in a subset of children at increased risk for recurrence/progression. Efforts to risk stratify children with type I PPB to optimize outcomes while reducing treatment-related side effects are underway.

Impact of the COVID-19 Pandemic on Utilization and Cost for Care of Pediatric ALL.

Objective: Acute lymphoblastic leukemia (ALL) is the most common childhood malignancy and requires a unique pattern of healthcare utilization including an acute/emergent presentation and an intensive initial 8 months of therapy followed by two years of outpatient treatment. The COVID-19 pandemic caused massive global disruptions in healthcare use and delivery. This report aims to examine the effects of the COVID-19 pandemic on the presentation, diagnosis and continued management of childhood ALL in regard to utilization and cost of care. Results: Utilizing a commercial insurance claims database, 529 pediatric patients were identified who were diagnosed with ALL and completed their initial 8 months of treatment between January 2016 and December 2021. New diagnoses were evaluated by era and demographics. Utilization was measured by COVID-related era as number of inpatient and outpatient encounters, inpatient days, and cumulative cost. None of these cost or utilization factors changed significantly during or shortly after the pandemic. These findings reinforce that the necessary care for pediatric ALL is largely inflexible and was unwavering despite the massive shifts in the healthcare system caused by the COVID-19 pandemic. This provides a valuable benchmark as we further examine the factors that influence the pandemic's impact on health equity and access to care, especially in vulnerable pediatric populations. This is the first investigation of the effect of the COVID-19 pandemic on utilization and cost of care in pediatric cancer.

Real-World Cost of Pediatric Acute Lymphoblastic Leukemia Care Among Commercially Insured Individuals in the United States: Effect of Era and Age at Diagnosis.

PURPOSE: Acute lymphoblastic leukemia (ALL) is the most common pediatric malignancy. Five-year survival is approaching 90%. In efforts to further improve outcomes, it is critical to consider the cost of ALL care. MATERIALS AND METHODS: Commercial insurance data from OptumLabs Data Warehouse were used to identify patients with ALL, age 1-30 years, diagnosed in 1993-2017 in the United States, with 36 months of continuous insurance coverage. Patients treated with hematopoietic cell transplantation were excluded. Inpatient and outpatient utilization and cumulative reimbursements (inflation-adjusted to December 2020) were computed 8 and 36 months from diagnosis and stratified by age (1-9, 10-12, and ≥ 13 years) as proxies for National Cancer Institute risk groups. Regression models were constructed to assess associations with demographic and clinical characteristics. RESULTS: Among 927 patients (median age, 6 years; interquartile range, 3-12 years; 43% female), individuals age ≥ 10 years had 23-25 more inpatient days and 22 more outpatient encounters compared with younger patients. The 36-month median cost was $394,000 (USD) (interquartile range, $256,000-$695,000 [USD]), and 64% of the total cost was incurred during the initial 8 months. The 36-month cost was 1.5-fold higher for those age 10-12 years and 1.7-fold higher for those age ≥ 13 years compared with 1-9 years. The cost for those diagnosed in 2013-2017 was 70% higher compared with 1993-2002, and was not different on the basis of sex, race, or ethnicity. CONCLUSION: Older age was associated with higher utilization and cost, and the cost of treatment increased significantly over time. These data provide valuable benchmarks for future studies examining the cost-benefit of ALL therapy modifications.

Resuscitation With Ringer's Lactate Compared With Normal Saline for Pediatric Diabetic Ketoacidosis.

OBJECTIVE: The aims of this study were to describe the use of Ringer's lactate (LR) or normal saline (NS) for resuscitation among children with diabetic ketoacidosis (DKA) and compare the effect of fluid type on cost, length of stay, and rate of cerebral edema (CE). METHODS: This is a retrospective study of 49,737 children aged 0 to 17 years with DKA between January 1, 2005, and September 30, 2015, using data from the Pediatric Health Information System. Treatment with LR or NS was identified. Our primary outcomes were total adjusted cost and length of stay. Our secondary outcome was CE rate per 1000 episodes. RESULTS: The majority of patients were treated with NS (n = 43,841 [88%]) compared with LR (n = 1762 [4%]) or both NS and LR (n = 4134 [8%]). Hospital-year-specific practice patterns were used to investigate the effect of fluid type across resuscitation fluid groups. Total adjusted cost was $1160 less (95% confidence interval, -1468 to -852), or -14.2%, for cases with any episode of LR compared with NS only. Length of stay was not different across groups. The rate of cerebral edema per 1000 episodes was 12.7 for cases with any episode of LR compared with 34.6 NS only (difference, -21.9; 95% confidence interval, -30.4 to -13.3). CONCLUSIONS: Ringer's lactate was infrequently used for resuscitation of pediatric DKA patients. However, resuscitation with LR compared with NS was associated with lower total cost and rates of CE. Further investigation using patient-level clinical and laboratory data is needed to evaluate factors that drive cost and risk of CE development with each fluid.

Pleuropulmonary blastoma-like peritoneal sarcoma: a newly described malignancy associated with biallelic DICER1 pathogenic variation.

Since the original description of pathogenic germline DICER1 variation underlying pleuropulmonary blastoma (PPB), the spectrum of extrapulmonary neoplasms known to be associated with DICER1 has continued to expand and now includes tumors of the ovary, thyroid, kidney, eye, and brain among other sites. This report documents our experience with another manifestation: a primitive sarcoma that resembles PPB and DICER1-associated sarcoma of the kidney. These tumors are distinguished by their unusual location in the peritoneal cavity, associated with visceral and/or parietal mesothelium. A total of seven cases were identified through pathology review in children presenting at a median age of 13 years (range 3-14 years). Primary sites of origin included the fallopian tube (four cases), serosal surface of the colon (one case), and pelvic sidewall (two cases). One case had pathologic features of type I PPB, another type Ir (regressed) PPB, and the remaining five had features of type II or III PPB with a mixed primitive sarcomatous pattern with or without cystic elements. All had a pathogenic DICER1 variation identified in germline and/or tumor DNA. PPB-like peritoneal tumors represent a newly described manifestation of DICER1 pathogenic variation whose pathologic features are also recapitulated in DICER1-related renal sarcoma, cervical embryonal rhabdomyosarcoma, and some Sertoli-Leydig cell tumors with heterologous elements. Tumors arising from the fallopian tube or elsewhere in the abdomen/pelvis, especially those with heterogeneous rhabdomyosarcomatous and/or cartilaginous differentiation, should prompt consideration of germline and tumor DICER1 testing.

Potentially inappropriate medication use by Medicaid+Choice beneficiaries in the last year of life.

BACKGROUND: Regardless of the payer and the period studied the prevalence of potentially inappropriate medication use in the elderly ranged from 21% to 40%. OBJECTIVE: To look at potentially inappropriate prescribing in a group of Medicare+Choice beneficiaries in their last year of life (LYOL) in a large national managed care organization. RESEARCH DESIGN: Retrospective review of Medicare+Choice decedents' drug claims and enrollment data collected between January 1998 and December 2000, supplemented by the Medicare denominator file and 1990 Census data. SUBJECTS: Four thousand six hundred two beneficiaries in a large national managed care organization. MEASURES: We analyzed the relationship between disagreement with the Beers' criteria and sociodemographic descriptors, insurance characteristics, and cause of death. We used logistic regression techniques to estimate factors associated with the disagreement. RESULTS: Two thousand thirty-one beneficiaries (44%) had at least one claim in the LYOL that disagreed with a Beers' criterion, 15% experienced more than one unique Beers' disagreement. The most common disagreements were for the use of propoxyphene (15.0%), followed by zolpidem (3.8%), and amitriptyline (2.8%). Based on total claims, cancer patients were most likely to receive propoxyphene (35.3%) followed by patients with a heart condition (29.6%). A large proportion of the potentially inappropriate prescribing involves psychoactive drugs. The logistic model showed fewer Beers' criteria breaches associated with being male and being non-white. Beers' breaches were more common if the beneficiary has increasing prescription use or died from cancer. CONCLUSION: This study showed that many beneficiaries have prescriptions that contravene the Beers' criteria.

Prescription drug spending for Medicare+Choice beneficiaries in the last year of life.

BACKGROUND: In 2006, Medicare implemented its prescription benefit plan. Therefore, insights into medication costs at the end of life may help guide clinicians to navigate Medicare Part D coverage for chronically ill individuals. OBJECTIVES: We examined drug spending by disease and demographics for Medicare+Choice (M+C) beneficiaries in the last year of life (LYOL). RESEARCH DESIGN: Retrospective review of M+C decedents' drug claims and enrollment data collected between January 1998 and December 2000, supplemented by the Medicare denominator file and 1990 Census data. SUBJECTS: Four thousand six hundred two beneficiaries in a large national managed care organization. MEASURES: We analyzed the relationship between prescription drug expenditures and sociodemographic descriptors, insurance characteristics, and cause of death. RESULTS: The mean annual number of prescriptions filled was 36.9; the managed care organization (MCO) paid $539 and beneficiaries paid $627. Higher expenditures were significantly correlated with female gender, higher number of comorbidities, and whether beneficiaries obtained the insurance as an employer-based retiree benefit. Minority beneficiaries had 26% fewer prescriptions. Increasing levels of annual median household income corresponded with a 20% increase in the number of prescriptions and a 25% increase in mean out-of-pocket expenses, between those with a median household income of less than $20,000 and those with $40,000 or greater. In the LYOL, chronic obstructive pulmonary disease and diabetes had the highest average number of prescriptions and total expenditures. Individuals dying from strokes or other unclassifiable conditions had the lowest average number of prescriptions and average total expenditures. CONCLUSION: Medication expenditures in the LYOL were highly dependent upon selected sociodemographic, insurance characteristics, and disease states.