Avery A Rizio1, Michelle K White2, Kristen L McCausland3, Tiffany P Quock4, Spencer D Guthrie5, Miyo Yokota6, Martha S Bayliss7. 1. Scientist, Optum, Johnston, RI. 2. Senior Scientist, Optum. 3. Scientist, Optum. 4. Senior Medical Director, Head of Medical Affairs, Prothena Biosciences, South San Francisco, CA. 5. Vice President, Global TTR Strategy, Akcea Therapeutics, San Francisco, CA. 6. Professor, National Institute of Technology-Hachinohe College, Japan. 7. Principal Consultant & Vice President of Patient Insights, Optum.
Abstract
BACKGROUND: Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment. OBJECTIVES: To explore the prevalence of AEs and treatment tolerability problems as reported by patients who received therapies for AL amyloidosis, and to examine the effects of AEs on treatment continuation and on health-related quality of life (HRQOL). METHODS: Patients with AL amyloidosis were recruited for this noninterventional, longitudinal, online survey. The patients responded to survey items regarding demographics, disease characteristics, most recent AL amyloidosis treatment, and HRQOL. The study analyses are based on data collected during the 6-month follow-up survey and are restricted to patients who completed the baseline and 6-month surveys and received treatment for AL amyloidosis within 6 months before the follow-up survey. RESULTS: A total of 100 patients met the inclusion criteria and were included in the study. The patients self-reported having a variety of AEs, which ranged in severity. Overall, 69.4% of patients had problems tolerating their treatment in the past 6 months, of whom 22% discontinued at least 1 therapy. In addition, approximately 33% of patients reduced their AL amyloidosis treatment because of AEs. Most often reported AEs included fatigue (83%), shortness of breath (53%), nausea (52%), and diarrhea (51%). Overall, 50% of the patients reported that their treatment was moderately well-tolerated and 41% said it was very well-tolerated. Those whose treatment was not well-tolerated had significantly worse HRQOL than patients whose treatment was well-tolerated. CONCLUSIONS: Patient-reported experiences should be considered by clinicians when making treatment-related decisions. More research is needed to explore additional factors that may contribute to treatment discontinuation in patients with AL amyloidosis.
BACKGROUND: Immunoglobulin light-chain amyloidosis (AL amyloidosis) is a rare and often fatal disease for which there is currently no treatment approved by the US Food and Drug Administration or the European Medicines Agency. Treatment options, which are typically based on therapies for multiple myeloma and are used off-label, are associated with substantial adverse events (AEs). Because the severity of AEs is often determined by clinicians, evaluations of treatment tolerability may not fully consider patients' own experience with treatment. OBJECTIVES: To explore the prevalence of AEs and treatment tolerability problems as reported by patients who received therapies for AL amyloidosis, and to examine the effects of AEs on treatment continuation and on health-related quality of life (HRQOL). METHODS: Patients with AL amyloidosis were recruited for this noninterventional, longitudinal, online survey. The patients responded to survey items regarding demographics, disease characteristics, most recent AL amyloidosis treatment, and HRQOL. The study analyses are based on data collected during the 6-month follow-up survey and are restricted to patients who completed the baseline and 6-month surveys and received treatment for AL amyloidosis within 6 months before the follow-up survey. RESULTS: A total of 100 patients met the inclusion criteria and were included in the study. The patients self-reported having a variety of AEs, which ranged in severity. Overall, 69.4% of patients had problems tolerating their treatment in the past 6 months, of whom 22% discontinued at least 1 therapy. In addition, approximately 33% of patients reduced their AL amyloidosis treatment because of AEs. Most often reported AEs included fatigue (83%), shortness of breath (53%), nausea (52%), and diarrhea (51%). Overall, 50% of the patients reported that their treatment was moderately well-tolerated and 41% said it was very well-tolerated. Those whose treatment was not well-tolerated had significantly worse HRQOL than patients whose treatment was well-tolerated. CONCLUSIONS: Patient-reported experiences should be considered by clinicians when making treatment-related decisions. More research is needed to explore additional factors that may contribute to treatment discontinuation in patients with AL amyloidosis.
Authors: R L Comenzo; V Sanchorawala; C Fisher; G Akpek; M Farhat; S Cerda; J L Berk; L M Dember; R Falk; K Finn; M Skinner; E Vosburgh Journal: Br J Haematol Date: 1999-03 Impact factor: 6.998
Authors: Sumit Madan; Shaji K Kumar; Angela Dispenzieri; Martha Q Lacy; Suzanne R Hayman; Francis K Buadi; David Dingli; S Vincent Rajkumar; William J Hogan; Nelson Leung; Martha Grogan; Morie A Gertz Journal: Blood Date: 2011-12-06 Impact factor: 22.113
Authors: Richard Specter; Vaishali Sanchorawala; David C Seldin; Anthony Shelton; Salli Fennessey; Kathleen T Finn; Jerome B Zeldis; Laura M Dember Journal: Nephrol Dial Transplant Date: 2010-08-05 Impact factor: 5.992
Authors: Arnaud Jaccard; Philippe Moreau; Veronique Leblond; Xavier Leleu; Lotfi Benboubker; Olivier Hermine; Christian Recher; Bouchra Asli; Bruno Lioure; Bruno Royer; Fabrice Jardin; Frank Bridoux; Bernard Grosbois; Jérome Jaubert; Jean-Charles Piette; Pierre Ronco; Fabrice Quet; Michel Cogne; Jean-Paul Fermand Journal: N Engl J Med Date: 2007-09-13 Impact factor: 91.245
Authors: Angela Dispenzieri; Martha Q Lacy; S Vincent Rajkumar; Susan M Geyer; Thomas E Witzig; Rafael Fonseca; John A Lust; Philip R Greipp; Robert A Kyle; Morie A Gertz Journal: Amyloid Date: 2003-12 Impact factor: 7.141