Huamao Mark Lin

The patient’s perspective on the symptom and everyday life impact of AL amyloidosis

Abstract Introduction: This study aimed to understand the symptomatic impact of amyloid light-chain (AL) amyloidosis from the patient’s perspective. Methods: Four data sources were included: a literature review, review of online patient blogs, expert clinician interviews and patient interviews. Patients were recruited through the Amyloidosis Foundation and physician referral. Phone interviews were conducted and included open-ended concept elicitation questions. Thematic analysis was performed to identify symptoms and impacts. Descriptive statistics were used to characterize the sample. A conceptual model was developed depicting the impact of disease and treatment. Results: Two hundred seventy abstracts were identified; 10 articles were deemed relevant. No qualitative studies were identified, and only three studies included patient-reported measures. Ten patients completed interviews (mean age 61 [±8]; 7 male). Over 25 signs/symptoms were identified, including fatigue, weakness, dyspnea, neuropathy, edema, dizziness/lightheadedness, anorexia, diarrhea and constipation. Impacts included reduced physical and social functioning, and emotional impacts, including frustration, anxiety and depression. Findings from the blogs and expert interviews were consistent with patient reports. Conclusion: Symptoms can vary widely, but a core set of symptoms were common across patients. The conceptual model derived from this study can be used to ensure a patient-centered approach to drug development.

Treatment patterns and health care resource utilization among patients with relapsed/refractory systemic light chain amyloidosis

Abstract Background: Treatment for patients with systemic light chain (AL) amyloidosis remains challenging. Our study aims to describe treatment patterns for both newly diagnosed and relapsed/refractory AL (RRAL) amyloidosis, and to assess clinical outcomes, healthcare costs, and resource utilization during the first year following a diagnosis of RRAL amyloidsis. Methods: This was a retrospective observational study of adult patients with AL amyloidosis using the US Optum administrative claims data during 1/1/2008 to 6/30/2015. Diagnosis was based on both ICD-9 codes and treatments with a claim for AL-amyloidosis-specific anticancer systemic agents. Results: Of 334 patients with AL amyloidosis, 43.1% were considered as RRAL amyloidosis. The majority (75%) of RRAL amyloidosis patients had organ involvement prior to the second line treatment. Proteasome-inhibitor-based regimens were most frequently used (41.0% for first-line AL, 30.6% for RRAL amyloidosis). Organ deterioration and mortality rates were 49.3% and 10.4%, respectively, during the two years following relapse. The average monthly cost was

Disease burden of systemic light-chain amyloidosis: a systematic literature review

14,369 per patient for RRAL amyloidosis including medical costs (

Patient-reported health-related quality of life from the phase III TOURMALINE-MM1 study of ixazomib-lenalidomide-dexamethasone versus placebo-lenalidomide-dexamethasone in relapsed/refractory multiple myeloma

9441) and drug costs (

Clinical and economic burden of peripheral T-cell lymphoma in commercially insured patients in the United States: findings using real-world claims data.

4928). Conclusions: RRAL amyloidosis is associated with high morbidity from target organ failure and mortality, which emphasizes the need for novel medications to improve care for patients with RRAL amyloidosis.

Healthcare resource utilization with ixazomib or placebo plus lenalidomide-dexamethasone in the randomized, double-blind, phase 3 TOURMALINE-MM1 study in relapsed/refractory multiple myeloma

Abstract Introduction: A systematic literature review on systemic light chain (AL) amyloidosis was conducted in order to understand the disease burden, and identify unmet medical needs and knowledge gaps. Methods: MEDLINE, Embase and Cochrane databases were searched for English language studies published in the last 10 years using search terms that focused on the clinical, economic, and patient-reported outcome (PRO) aspects of AL amyloidosis. There was a low yield of articles in the economic and PRO categories and additional searches were conducted in clinical conference proceedings, and using Google and Google Scholar. After review, there were 65 articles included for data extraction. Results: AL amyloidosis is a rare disorder without any FDA or EMA approved indications for drug therapy. Using off-label therapies, there is a high rate, 42–64%, of non-response or progression, and an associated high mortality. Toxicities during therapy are common with estimates of up to 30–40% of patients experiencing severity of grade 3 or higher. Patients with AL amyloidosis report severe psychological distress, anxiety and clinical depression. Conclusions: There is a deficiency in the literature on the economic costs associated with AL amyloidosis, and information on costs has been derived from studies that examined multiple myeloma or other disease or treatment components common to AL amyloidosis.

Real-World Treatment Patterns and Health Care Resource Utilization in Relapsed or Refractory Multiple Myeloma: Evidence from a Cohort Review in France

TOURMALINE‐MM1 is a phase III, randomized, double‐blind, placebo‐controlled study of ixazomib plus lenalidomide and dexamethasone (IRd) versus placebo‐Rd in patients with relapsed/refractory multiple myeloma following 1–3 prior lines of therapy. The study met its primary endpoint, demonstrating significantly longer progression‐free survival (PFS) in the IRd arm versus placebo‐Rd arm (median 20.6 vs 14.7 months, hazard ratio 0.74, P = .01), with limited additional toxicity. Patient‐reported health‐related quality of life (HRQoL) was a secondary endpoint of TOURMALINE‐MM1. The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core‐30 (QLQ‐C30) and Multiple Myeloma Module 20 (QLQ‐MY20) were completed at screening, the start of cycles 1 and 2, every other cycle, the end of treatment, and every 4 weeks until progression. Over median follow‐up of 23.3 and 22.9 months in the IRd and placebo‐Rd arms, mean QLQ‐C30 global health status (GHS)/QoL scores were maintained from baseline over the course of treatment in both groups, with no statistically significant differences between groups. EORTC QLQ‐C30 function domain scores were also generally maintained from baseline; similarly, physical, emotional, and social function domains were maintained with IRd versus placebo‐Rd, with slightly higher mean change from baseline scores at earlier time points with IRd. Findings from this double‐blind study demonstrate that addition of ixazomib to Rd significantly improved efficacy while HRQoL was maintained, reflecting the limited additional toxicity seen with IRd versus placebo‐Rd, and support the feasibility of long‐term IRd administration.

Multiple Myeloma Patients’ Profile and Regimens Transitions in the Brazilian Private Health Sector

Abstract Objective: This retrospective cohort study utilized real-world claims data to assess the clinical and economic burden of peripheral T-cell lymphoma (PTCL) over the continuum of care in the US. Methods: Data were extracted from US administrative claims databases to identify adult patients with PTCL (ICD-9-CM code 202.7X) diagnosed between October 2007 and June 2011. Patients had to have ≥6 months of continuous enrollment before and ≥12 months of continuous enrollment after their index date (date of first PTCL diagnosis). PTCL patients were matched (1:5) by age, sex, region, plan type, payer type, and length of continuous enrollment, to a control group of randomly selected patients without PTCL. Patient-level healthcare resource utilization data and associated costs (in US dollars) were measured. Mean costs per patient per month were determined. Results: Of 2820 patients with PTCL, 1000 met all inclusion criteria (median age = 57 years; 57.5% male) and were matched to the control group (n = 5000). On an average monthly basis, PTCL patients were hospitalized more frequently (0.07 vs 0.01 admissions; p < 0.0001) and had a longer length of hospital stay (6.4 vs 4.0 days; p < 0.0001) compared with controls. PTCL patients also had higher monthly utilization of pharmacy services (2.85 vs 0.97 prescriptions; p < 0.0001), office visits (1.35 vs 0.34 visits; p < 0.0001), ER visits (0.07 vs 0.02 visits; p < 0.0001), hospice stays (0.05 vs 0.01 stays; p < 0.0001) and other patient services/procedures. Overall, PTCL patients incurred higher average monthly costs per patient compared with control patients (

Real-World Treatment Patterns in Relapsed or Refractory Multiple Myeloma: Evidence from a Medical Chart Review in the United Kingdom

6327.84 vs

Real-World Treatment Patterns and Health Care Resource Utilization in Relapsed or Refractory Multiple Myeloma: Evidence from a Medical Chart Review in France

388.39; p < 0.0001), driven mainly by hospitalizations (32.2% of overall costs) and pharmacy services (19.6%). Conclusions: This is the first real-world study to quantify healthcare resource utilization, costly treatment, and overall medical expenditure in commercially insured PTCL patients. Better tolerated and more effective treatments may improve disease management and reduce the clinical and economic burden of PTCL.