Purpose:The aim of the study was to project the long-term net health benefits of mavacamten for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM) in the United States.Methods: A Markov model with 4 mutually exclusive health states (New York Heart Association [NYHA] functional classes I, II, and III/IV and death) was developed to project the life-years (LYs) and quality-adjusted life-years (QALYs) over a lifetime horizon for patients with symptomatic obstructive HCM receiving mavacamten with or without βblocker (BB) or calcium channel blocker (CCB) monotherapy or placebo with or without BB or CCB monotherapy. The model simulated a patient cohort with a starting age of 59 years and 41% women. Transition probabilities across NYHA functional classes were estimated using data from the Phase III Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy (EXPLORER-HCM) and the EXPLORER long-term extension (EXPLORER-LTE) cohort from the Long-term Safety Extension Study of Mavacamten in Adults who Have Completed MAVERICK-HCM or EXPLORER-HCM (MAVA-LTE) trial and were extrapolated after week 30. The mortality risks of NYHA functional class I were assumed to be the age-and sex-specific mortality risks of the US general population. The mortality risks for NYHA class II and III/IV were estimated using those

Aims: Obstructive hypertrophic cardiomyopathy (oHCM) is a disease of the cardiomyocyte in which dynamic left ventricular outflow track obstruction may lead to heart failure, valvular disease, and sudden death. Little is known about healthcare resource utilization (HRU) and costs associated with oHCM. This study investigated the clinical and economic burden of oHCM in patients with or without symptoms associated with oHCM. Methods:We used the US IBM® MarketScan® Commercial and Medicare Supplemental database to identify patients with oHCM (January 2009-March 2019). Control patients without cardiomyopathy were matched to each patient with oHCM based on age, sex, region, and index year (3:1 ratio). One-year HRU and cost data were compared between all oHCM, symptomatic oHCM, and asymptomatic oHCM subgroups, and their respective controls.Results: Among 11,401 eligible patients with oHCM (mean age 57 years, 42% female), 5,667 (50%) were symptomatic (23% chest pain, 57% dyspnea, 29% fatigue, 17% syncope). oHCM was associated with significant increases in all-cause hospitalizations, hospital days, outpatient visits, and total healthcare costs (mean ± standard deviation: $26,929±77,720 vs. $6,808±25,712, p<.001) compared with matched controls. These differences were driven mainly by the clinical and economic burden of symptomatic oHCM, which was associated with significant increases in 1-year hospitalization rates (38.0% vs. 6.9%), hospital days (3.7±9.9 vs. 0.4±3.0), and total healthcare costs ($43,586±103,756 vs. $6,768±27,618; all p<.001). Adjustment for comorbidities had minimal impact on these differences. Limitations:The use of claims data relies on International Classification of Diseases (ICD-9 and ICD-10) diagnosis codes, which might be inaccurate. Only commercially insured patients were included. Conclusion:In a real-world population, oHCM was associated with substantial increases in HRU and incremental

BackgroundTotal skin electron beam radiation therapy (TSEBT) is an effective treatment for primary diffuse cutaneous lymphomas in humans. While several techniques exist, they all require significant commitment of staff time and resources. In veterinary medicine, canine-specific techniques and strategies have been adapted and delivered but deemed not “realistically” clinically implementable given the time commitment of over 2.5 h plus per fraction or have been relegated to palliative intent. Leveraging these technologies of helical tomotherapy and 3D printing, we developed and clinically implemented a radiotherapeutic treatment strategy for the management of medically refractory diffuse cutaneous lymphoma in the dog.Case presentationA 13.5-year-old female spayed Bichon Frise presented to the Oncology service at Texas A&M University, College of Veterinary Medicine due to the progression of diffuse cutaneous epitheliotropic lymphoma (CEL) that had failed medical management. Twenty-seven gray were delivered to the patient with a treatment time requirement under 40 min including real time monitoring of anesthesia during setup and treatment. A partial response was noticeable after four fractions and the tumor completely regressed progressively over the entire treated area by the end of therapy. A grade 1 lethargy, fatigue, weight loss, and oral mucositis and grade 2 alopecia, nail/claw changes, pruritus, scaling, anorexia, and diarrhea were noted during treatment. Additionally, a grade 3 thrombocytopenia developed after fraction eight requiring a treatment interruption of 6 weeks and prescription modification prior to treatment continuation and completion. From the beginning of total skin photon radiation therapy (TSPT) treatment until the time of the patient was euthanized unrelated to cutaneous epitheliotropic lymphoma (123 days), only one new lesion on the head was identified and confirmed by histopathology within the treated fields.ConclusionsThe proposed technique is an acceptable alternative to TSEBT that is actually clinically implementable within a palliative or definitive setting and clinical constraints, however further testing and refinement is needed to reduce hematological complications and to confirm and expand on preliminary findings.

Introduction: There is limited evidence on therapies for obstructive hypertrophic cardiomyopathy (HCM), and data regarding treatment patterns and cost are scarce. This study assessed treatment patterns and economic outcomes in patients with symptomatic obstructive HCM.Methods: Adults with symptomatic obstructive HCM as per study design and treated with pharmacotherapies [beta blockers (BBs), calcium channel blockers (CCBs), BB ? CCB, or disopyramide] or procedures (septal reduction therapy, heart transplantation, implantable cardioverter-defibrillator, and pacemaker implantation) were identified from the IBMÒ MarketScanÒ Commercial and Medicare Supplemental database (January 2009-March 2019). Patients had 12-month continuous eligibility before and after (study period) treatment initiation (index treatment). Healthcare resource utilization (HRU), costs, and treatment changes were assessed. Results: Of the 4883 patients included in the analysis, 85% received pharmacotherapies (BB 52.5%; CCB 11.7%; BB ? CCB 17.7%; disopyramide 2.4%) and 15.7% underwent procedures. During the study period, 38, 34, and 100% of all patients had C 1 inpatient stay, emergency room (ER) visit, and outpatient visit, respectively; mean total healthcare costs were US$53,053. Patients undergoing procedures had the highest HRU and costs across groups. Among patients receiving pharmacotherapies, HRU was lowest with BBs and highest with disopyramide.Treatment changes were observed in 43.8% of patients receiving pharmacotherapies. Conclusions: Patients experienced high rates of treatment changes, and the economic burden associated with symptomatic obstructive HCM

Introduction: Mavacamten improved functional capacity and LVOT obstruction in patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in the EXPLORER-HCM study (NCT03470545). Objective: To determine the number needed to treat (NNT) to achieve a clinical response comparing 30-week treatment with mavacamten versus placebo in patients with symptomatic oHCM. Methods: Data from the EXPLORER-HCM trial were analyzed. Most patients (92%) were receiving beta-blockers or calcium channel blockers. Responses were assessed using several measures: primary efficacy endpoint (improvement of ≥1.5 mL/kg/min in pVO 2 and improvement of ≥1 NYHA class or improvement of ≥3.0 mL/kg/min in pVO 2 and no worsening in NYHA class); symptoms (NYHA class); PROs (Kansas City Cardiomyopathy Questionnaire Clinical Summary Score [KCCQ CSS] and Hypertrophic Cardiomyopathy Symptom Questionnaire Shortness of Breath [HCMSQ SoB]); hemodynamics (resting and post-exercise LVOT gradients); exercise capacity (pVO 2 ); biomarkers (NT-proBNP and hs-cTnI). NNT comparing mavacamten versus placebo was calculated as the reciprocal of the difference in response rates between the two groups at week 30 rounded up to an integer. Results (Table): The NNT to achieve the primary endpoint was 6. The NNTs were 3 for an improvement of ≥1 NYHA class and 4 for an improvement to NYHA class I. For PROs, the NNTs were 5 for an increase of ≥10 points in KCCQ CSS and 4 for a reduction of ≥2.5 points in HCMSQ SoB, which are considered clinically important changes. For hemodynamics, the NNTs were 2–3. For increases in pVO 2 ≥1.5 mL/kg/min and ≥3.0 mL/kg/min, the NNTs were 6 and 9, respectively. For biomarkers, the NNTs were 3 for any reduction in NT-proBNP and 5 for any reduction in hs-cTnI. Conclusions: The NNTs comparing mavacamten with placebo were consistently low across several clinical and PRO measures. This suggests that mavacamten may be an effective therapy for the treatment of symptomatic oHCM.