PMID- 30647830
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20220331
IS  - 1942-2962 (Print)
IS  - 1942-2970 (Electronic)
IS  - 1942-2962 (Linking)
VI  - 11
IP  - 8
DP  - 2018 Nov
TI  - Treatment Tolerability in Patients with Immunoglobulin Light-Chain Amyloidosis.
PG  - 430-437
AB  - 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.
FAU - Rizio, Avery A
AU  - Rizio AA
AD  - Scientist, Optum, Johnston, RI.
FAU - White, Michelle K
AU  - White MK
AD  - Senior Scientist, Optum.
FAU - McCausland, Kristen L
AU  - McCausland KL
AD  - Scientist, Optum.
FAU - Quock, Tiffany P
AU  - Quock TP
AD  - Senior Medical Director, Head of Medical Affairs, Prothena Biosciences, South San 
      Francisco, CA.
FAU - Guthrie, Spencer D
AU  - Guthrie SD
AD  - Vice President, Global TTR Strategy, Akcea Therapeutics, San Francisco, CA.
FAU - Yokota, Miyo
AU  - Yokota M
AD  - Professor, National Institute of Technology-Hachinohe College, Japan.
FAU - Bayliss, Martha S
AU  - Bayliss MS
AD  - Principal Consultant & Vice President of Patient Insights, Optum.
LA  - eng
PT  - Journal Article
PL  - United States
TA  - Am Health Drug Benefits
JT  - American health & drug benefits
JID - 101479877
PMC - PMC6306096
OTO - NOTNLM
OT  - adverse events
OT  - health-related quality of life
OT  - immunoglobulin light-chain amyloidosis
OT  - treatment discontinuation
OT  - treatment tolerability
EDAT- 2019/01/17 06:00
MHDA- 2019/01/17 06:01
PMCR- 2018/11/01
CRDT- 2019/01/17 06:00
PHST- 2019/01/17 06:00 [entrez]
PHST- 2019/01/17 06:00 [pubmed]
PHST- 2019/01/17 06:01 [medline]
PHST- 2018/11/01 00:00 [pmc-release]
PST - ppublish
SO  - Am Health Drug Benefits. 2018 Nov;11(8):430-437.