Survival of patients with chronic acid sphingomyelinase deficiency (ASMD) in the United States: A retrospective chart review study.

Background: Acid sphingomyelinase deficiency (ASMD), historically known as Niemann-Pick disease type A, A/B, and B, is a rare lysosomal storage pathology with multisystemic clinical manifestations. The aims of this study were to estimate the survival probability in patients in the United States with chronic ASMD (ASMD types B and A/B), and to describe the disease characteristics of these patients. Methods: This observational retrospective study included medical chart records of patients with chronic ASMD with retrievable data abstracted by 69 participating physicians from 25 medical centers in the United States. Included patients had a date of ASMD diagnosis or first presentation to a physician for ASMD symptoms (whichever occurred first) between January 01, 1990, and February 28, 2021. Medical chart records were excluded if patients were diagnosed with ASMD type A. Eligible medical chart records were abstracted to collect demographic, medical and developmental history, and mortality data. Survival outcomes were analyzed using Kaplan-Meier survival analyses from birth until death. Results: The overall study population (N = 110) included 69 patients with ASMD type B, nine with type A/B, and 32 with ASMD "non-type A" (ASMD subtype was unknown, but patients were confirmed as not having ASMD type A). The majority of patients were male with a median age at diagnosis of 3.8 years. Thirty-eight patients died during the study observation period, at a median age of 6.8 years. The median (95% confidence interval) survival age from birth was 21.3 (10.2; 60.4) years. At diagnosis or first presentation, 42.7% patients had ≥1 ASMD-related complication; splenic (30.0%) and hepatobiliary (20.9%) being the most common, and 40.9% required ≥1 medical visit due to complications. Conclusion: Patients with chronic ASMD in the United States have poor survival and significant burden of illness.

Budget impact analysis of belumosudil for chronic graft-versus-host disease treatment in the United States.

AIMS: To assess the impact of belumosudil on the cost of care in chronic graft-versus-host disease (cGVHD) patients who have failed at least two prior lines of systemic therapy using a budget impact model. METHODS: A budget impact model with a 5-year time horizon was constructed in Microsoft Excel. The base case model uses the US prevalence rate of 3 L/4L + cGVHD patients from literature and secondary sources, with the potential for user-defined inputs, including model perspectives. The model includes data for two perspectives: the national US population and a hypothetical US private payer health insurance plan with 10 million (Mil) members. Additional model inputs include market share of cGVHD treatments, their associated adverse event rates, and healthcare resource utilization. RESULTS: The potential annual budget impact for the US national and payer plans was evaluated for cGVHD patients. Based on belumosudil utilization increasing to 55% in 3 L and 4 L + by 2026, cost savings of ∼5.5% and 6.7% ($128.8 and $4.9 Mil USD) were observed from national and payer perspectives, respectively. Cost savings in 2026 were derived from fewer AEs ($108.4 and $3.9 Mil USD, for national and payer perspectives; e.g. neutropenia, and thrombocytopenia) and reduced HCRU ($65.1 and $2.3 Mil USD, for national and payer perspectives; e.g. emergency room visits, ICU stays, etc.). LIMITATIONS: Results from the model were dependent on the available data inputs and assumptions. Real-world values may differ from the assumed performance of treatments, market growth, and treatment dosing and duration. CONCLUSION: The model results suggest that the introduction of belumosudil to treat cGVHD would be associated with substantial cost savings when evaluating a scenario with versus without belumosudil from a US payer perspective.

Epidemiology and Treatment of Chronic Graft-versus-Host Disease Post-Allogeneic Hematopoietic Cell Transplantation: A US Claims Analysis.

Chronic graft-versus-host disease (cGVHD) is a complication of hematopoietic cell transplantation (HCT). Although the clinical outcomes of cGVHD are well documented, few studies have assessed treatment practices outside of clinical trials. The present study aimed to quantify the prevalence of cGVHD, examine provider prescribing patterns, and evaluate the healthcare cost and resource utilization (HCRU) in a US cGVHD population. We analyzed anonymized claims from the Medicare Fee-for-Service (FFS) 5% sample for beneficiaries enrolled between 2013 and 2016 and PharMetrics commercial 2013 to 2018 databases to identify cGVHD in allogeneic HCT recipients. cGVHD was identified based on International Classification of Diseases Ninth/Tenth Revision diagnosis codes for cGVHD or unspecified GVHD with a first diagnosis >180 days post-HCT or a maintained unspecified GVHD diagnosis for >12 months postindex of unspecified GVHD diagnosis. Longitudinal and line of therapy (LOT) analyses were based on the PharMetrics dataset for 2013 to 2018. Healthcare costs were calculated by adding the inpatient, outpatient, and pharmacy insurer and beneficiary paid amounts for the commercially insured population. Total HCRU was assessed using the number of inpatient and outpatient visits following the initial cGVHD diagnosis. In 2016, the projected prevalence of cGVHD in the United States based on the Medicare FFS and PharMetrics commercial databases was 14,017 individual patients. Within 3 years after undergoing allogeneic HCT, 42% of patients developed cGVHD; 66% of the cGVHD patients had a prior diagnosis of acute GVHD. The majority of cGVHD patients received at least one systemic therapy; 71% and 47% of cGVHD patients progressed to a second and third LOT, respectively. A total of 24 unique therapeutic agents and more than 150 combinations were used in the second and third LOTs. Corticosteroids and corticosteroid combination therapy were the most common forms of treatment across all examined LOTs. Furthermore, the most commonly used agents in the first LOT, second LOT, and third LOT were corticosteroids only, calcineurin inhibitors only, and corticosteroids only, respectively. In the 12 months postdiagnosis, cGVHD patients had an average of 21.0 cGVHD-related inpatient and outpatient visits (2.8 inpatient and 18.2 outpatient visits). A significant proportion of allogeneic HCT recipients continue to develop cGVHD, and despite advances in the understanding of cGVHD, corticosteroids remain the mainstay of therapy. Patients often progress beyond the first LOT, at which time the utilization of systemic therapies is highly variable, demonstrating the need for evidence-based treatment approaches.

Evaluation of Postgraduates Following Implementation of a Focus Assessed Transthoracic Echocardiography (FATE) Training Course-A Pilot Study.

At our institution, implementation of a formal training course in Basic Focus Assessed Transthoracic Echocardiography (FATE) was associated with an improvement in anesthesia trainees' ability to obtain transthoracic echocardiography (TTE) images. Total image acquisition scores improved by a median (Q1, Q3) 9.1 (2.9,14.7) percentage points from pre-to post-hands-on FATE course (n=20; p=0.001). Participants who returned for a subsequent assessment 5 months following the course demonstrated a median (Q1, Q3) 18.0 (9.1,22.1) percentage point improvement from their pre-course total image acquisition scores (n=11; p=0.002). This pilot study established the feasibility of our program and results suggest that the basic FATE course can be used to teach trainees TTE quickly, effectively, and with significant retention.

Prospective, Randomized Double-Blind Study: Does Decreasing Interscalene Nerve Block Volume for Surgical Anesthesia in Ambulatory Shoulder Surgery Offer Same-Day Patient Recovery Advantages?

BACKGROUND AND OBJECTIVES: In this randomized double-blind prospective study in patients undergoing shoulder arthroscopy, we compared the effects of ultrasound-guided interscalene nerve block using 20 mL (intervention group) and 40 mL (control group) of a mepivacaine 1.5% and bupivacaine 0.5% mixture (1:1 volume) on ipsilateral handgrip strength and other postoperative end points. METHODS: One hundred fifty-four patients scheduled for ambulatory shoulder arthroscopy were randomly assigned to receive a single-injection interscalene block under ultrasound guidance with either 40 mL (control) or 20 mL (intervention) and intravenous sedation. The primary outcome was the change in ipsilateral handgrip strength in the postanesthesia care unit (PACU) measured with a dynamometer. Secondary end points were recorded, including negative inspiratory force, incidences of hoarseness and Horner syndrome, time to readiness for discharge from PACU, time to discharge from PACU, patient satisfaction, time to block resolution, and pain scores. RESULTS: Postoperative handgrip strength was greater in the 20-mL group compared with the 40-mL group (difference in means, 2.3 kg [95% confidence interval, 0.6-4.0 kg]; P = 0.009). A smaller proportion of patients in the intervention group experienced hoarseness postoperatively compared with the control group (odds ratio, 0.26 [95% confidence interval, 0.08-0.82]; P = 0.015). Patient satisfaction and duration of analgesia were similar in both groups. CONCLUSIONS: When used for surgical anesthesia for shoulder arthroscopies in the ambulatory setting, a 20-mL volume in an ultrasound-guided interscalene block preserves greater handgrip strength on the ipsilateral side in the PACU compared with 40 mL without significant decrease in block success, duration of analgesia, and patient satisfaction.