TREATMENT HISTORY, TOLERABILITY AND IMPACT ON HEALTH-RELATED QUALITY OF LIFE IN AL AMYLOIDOSIS
(Abstract release date: 05/19/16)
EHA Library. Guthrie S. 06/09/16; 132994; E1445
Mr. Spencer Guthrie
Contributions
Contributions
Abstract
Abstract: E1445
Type: Eposter Presentation
Background
Light-chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs. There are currently no FDA- or EMA-approved medications indicated for AL amyloidosis; however, chemotherapy, stem cell transplants (SCT), and immunomodulary drugs can reduce the production of amyloid-forming light chains. All existing regimens have tolerability problems due to treatment-related symptoms (TRSs). The SF-36v2® Health Survey (SF-36v2) is a widely-used general health-related quality of life (HRQoL) survey that can be used to describe and quantify the impact of many diseases and treatments.
Aims
To describe the history of treatments, past and current TRSs, and impact on HRQoL among a diverse sample of individuals with AL amyloidosis.
Methods
We report baseline data from an online non-interventional study was initiated in 2015 among patients with self-reported AL amyloidosis (n=341). Patients reported their current and prior treatment for AL amyloidosis. Aspects of TRSs were captured based on the following: 1) lifetime history of TRSs (dichotomous variable); 2) consequence of TRSs (discontinuation of a treatment; reduction of a treatment; or maintenance of treatment despite TRSs); and 3) ability to tolerate the current AL amyloidosis treatment (based on a 4 point scale from “extremely poorly” to “very well”, higher scores indicate better tolerability). The prevalence of each treatment type and TRSs were estimated. The patients’ ability to tolerate the current treatment was evaluated in relation to specific medications and HRQoL (as measured by the SF-36v2® Health Survey Physical [PCS] and Mental [MCS] Component Summary scores) using chi square tests for categorical variables and t-tests and ANOVA for continuous measures.
Results
The most commonly reported treatments were dexamethasone (81% reported ever being treated, 52% reported as the current treatment), bortezomib (72% ever, 36% current), SCT (53% ever, 24% current), melphalan (47% ever, 15% current), cyclophosphamide (46% ever, 20% current), and lenalidomide (28% ever, 15% current). Many patients reported combination treatments, including cyclophosphamide+bortezomib+dexamethasome (CyBorD, 17% current). Half of the patients (51%) had received three or more different treatments. Nearly three-quarters (71%, n=226) reported ever having problems tolerating AL amyloidosis treatment, of which nearly half (47%, n=107) had discontinued at least one treatment. Nearly half (46%) of those currently being treated reported some tolerability issue (less than very good tolerability). Tolerability varied across the common treatments from a low of 3.22 (SD=0.90) for cyclophosphamide to a high of 3.61 (SD=0.52) for SCT. Problems with tolerating current medications corresponded with decrements in HRQoL (both MCS and PCS, p<0.001).
Conclusion
Lifetime history of TRSs was high. Discontinuation of life-saving AL amyloidosis treatments was fairly common, though most patients were able to tolerate their current regimen. The high prevalence of treatment discontinuation and history of multiple AL amyloidosis treatments suggests that physicians and patients try a variety of treatments to balance tolerability and efficacy. TRSs are associated with decrements in HRQoL, over and above the burden of AL amyloidosis. These findings highlight the importance of assessing HRQoL during treatment for AL amyloidosis to better understand tolerability, and the need for more treatment options for AL amyloidosis, particularly those with favorable tolerability. Study supported by: Prothena Biosciences Inc
Session topic: E-poster
Keyword(s): AL amyloidosis, Quality of life, Tolerance
Type: Eposter Presentation
Background
Light-chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs. There are currently no FDA- or EMA-approved medications indicated for AL amyloidosis; however, chemotherapy, stem cell transplants (SCT), and immunomodulary drugs can reduce the production of amyloid-forming light chains. All existing regimens have tolerability problems due to treatment-related symptoms (TRSs). The SF-36v2® Health Survey (SF-36v2) is a widely-used general health-related quality of life (HRQoL) survey that can be used to describe and quantify the impact of many diseases and treatments.
Aims
To describe the history of treatments, past and current TRSs, and impact on HRQoL among a diverse sample of individuals with AL amyloidosis.
Methods
We report baseline data from an online non-interventional study was initiated in 2015 among patients with self-reported AL amyloidosis (n=341). Patients reported their current and prior treatment for AL amyloidosis. Aspects of TRSs were captured based on the following: 1) lifetime history of TRSs (dichotomous variable); 2) consequence of TRSs (discontinuation of a treatment; reduction of a treatment; or maintenance of treatment despite TRSs); and 3) ability to tolerate the current AL amyloidosis treatment (based on a 4 point scale from “extremely poorly” to “very well”, higher scores indicate better tolerability). The prevalence of each treatment type and TRSs were estimated. The patients’ ability to tolerate the current treatment was evaluated in relation to specific medications and HRQoL (as measured by the SF-36v2® Health Survey Physical [PCS] and Mental [MCS] Component Summary scores) using chi square tests for categorical variables and t-tests and ANOVA for continuous measures.
Results
The most commonly reported treatments were dexamethasone (81% reported ever being treated, 52% reported as the current treatment), bortezomib (72% ever, 36% current), SCT (53% ever, 24% current), melphalan (47% ever, 15% current), cyclophosphamide (46% ever, 20% current), and lenalidomide (28% ever, 15% current). Many patients reported combination treatments, including cyclophosphamide+bortezomib+dexamethasome (CyBorD, 17% current). Half of the patients (51%) had received three or more different treatments. Nearly three-quarters (71%, n=226) reported ever having problems tolerating AL amyloidosis treatment, of which nearly half (47%, n=107) had discontinued at least one treatment. Nearly half (46%) of those currently being treated reported some tolerability issue (less than very good tolerability). Tolerability varied across the common treatments from a low of 3.22 (SD=0.90) for cyclophosphamide to a high of 3.61 (SD=0.52) for SCT. Problems with tolerating current medications corresponded with decrements in HRQoL (both MCS and PCS, p<0.001).
Conclusion
Lifetime history of TRSs was high. Discontinuation of life-saving AL amyloidosis treatments was fairly common, though most patients were able to tolerate their current regimen. The high prevalence of treatment discontinuation and history of multiple AL amyloidosis treatments suggests that physicians and patients try a variety of treatments to balance tolerability and efficacy. TRSs are associated with decrements in HRQoL, over and above the burden of AL amyloidosis. These findings highlight the importance of assessing HRQoL during treatment for AL amyloidosis to better understand tolerability, and the need for more treatment options for AL amyloidosis, particularly those with favorable tolerability. Study supported by: Prothena Biosciences Inc
Session topic: E-poster
Keyword(s): AL amyloidosis, Quality of life, Tolerance
Abstract: E1445
Type: Eposter Presentation
Background
Light-chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs. There are currently no FDA- or EMA-approved medications indicated for AL amyloidosis; however, chemotherapy, stem cell transplants (SCT), and immunomodulary drugs can reduce the production of amyloid-forming light chains. All existing regimens have tolerability problems due to treatment-related symptoms (TRSs). The SF-36v2® Health Survey (SF-36v2) is a widely-used general health-related quality of life (HRQoL) survey that can be used to describe and quantify the impact of many diseases and treatments.
Aims
To describe the history of treatments, past and current TRSs, and impact on HRQoL among a diverse sample of individuals with AL amyloidosis.
Methods
We report baseline data from an online non-interventional study was initiated in 2015 among patients with self-reported AL amyloidosis (n=341). Patients reported their current and prior treatment for AL amyloidosis. Aspects of TRSs were captured based on the following: 1) lifetime history of TRSs (dichotomous variable); 2) consequence of TRSs (discontinuation of a treatment; reduction of a treatment; or maintenance of treatment despite TRSs); and 3) ability to tolerate the current AL amyloidosis treatment (based on a 4 point scale from “extremely poorly” to “very well”, higher scores indicate better tolerability). The prevalence of each treatment type and TRSs were estimated. The patients’ ability to tolerate the current treatment was evaluated in relation to specific medications and HRQoL (as measured by the SF-36v2® Health Survey Physical [PCS] and Mental [MCS] Component Summary scores) using chi square tests for categorical variables and t-tests and ANOVA for continuous measures.
Results
The most commonly reported treatments were dexamethasone (81% reported ever being treated, 52% reported as the current treatment), bortezomib (72% ever, 36% current), SCT (53% ever, 24% current), melphalan (47% ever, 15% current), cyclophosphamide (46% ever, 20% current), and lenalidomide (28% ever, 15% current). Many patients reported combination treatments, including cyclophosphamide+bortezomib+dexamethasome (CyBorD, 17% current). Half of the patients (51%) had received three or more different treatments. Nearly three-quarters (71%, n=226) reported ever having problems tolerating AL amyloidosis treatment, of which nearly half (47%, n=107) had discontinued at least one treatment. Nearly half (46%) of those currently being treated reported some tolerability issue (less than very good tolerability). Tolerability varied across the common treatments from a low of 3.22 (SD=0.90) for cyclophosphamide to a high of 3.61 (SD=0.52) for SCT. Problems with tolerating current medications corresponded with decrements in HRQoL (both MCS and PCS, p<0.001).
Conclusion
Lifetime history of TRSs was high. Discontinuation of life-saving AL amyloidosis treatments was fairly common, though most patients were able to tolerate their current regimen. The high prevalence of treatment discontinuation and history of multiple AL amyloidosis treatments suggests that physicians and patients try a variety of treatments to balance tolerability and efficacy. TRSs are associated with decrements in HRQoL, over and above the burden of AL amyloidosis. These findings highlight the importance of assessing HRQoL during treatment for AL amyloidosis to better understand tolerability, and the need for more treatment options for AL amyloidosis, particularly those with favorable tolerability. Study supported by: Prothena Biosciences Inc
Session topic: E-poster
Keyword(s): AL amyloidosis, Quality of life, Tolerance
Type: Eposter Presentation
Background
Light-chain (AL) amyloidosis is a rare disease characterized by misfolded amyloid protein deposits in tissues and vital organs. There are currently no FDA- or EMA-approved medications indicated for AL amyloidosis; however, chemotherapy, stem cell transplants (SCT), and immunomodulary drugs can reduce the production of amyloid-forming light chains. All existing regimens have tolerability problems due to treatment-related symptoms (TRSs). The SF-36v2® Health Survey (SF-36v2) is a widely-used general health-related quality of life (HRQoL) survey that can be used to describe and quantify the impact of many diseases and treatments.
Aims
To describe the history of treatments, past and current TRSs, and impact on HRQoL among a diverse sample of individuals with AL amyloidosis.
Methods
We report baseline data from an online non-interventional study was initiated in 2015 among patients with self-reported AL amyloidosis (n=341). Patients reported their current and prior treatment for AL amyloidosis. Aspects of TRSs were captured based on the following: 1) lifetime history of TRSs (dichotomous variable); 2) consequence of TRSs (discontinuation of a treatment; reduction of a treatment; or maintenance of treatment despite TRSs); and 3) ability to tolerate the current AL amyloidosis treatment (based on a 4 point scale from “extremely poorly” to “very well”, higher scores indicate better tolerability). The prevalence of each treatment type and TRSs were estimated. The patients’ ability to tolerate the current treatment was evaluated in relation to specific medications and HRQoL (as measured by the SF-36v2® Health Survey Physical [PCS] and Mental [MCS] Component Summary scores) using chi square tests for categorical variables and t-tests and ANOVA for continuous measures.
Results
The most commonly reported treatments were dexamethasone (81% reported ever being treated, 52% reported as the current treatment), bortezomib (72% ever, 36% current), SCT (53% ever, 24% current), melphalan (47% ever, 15% current), cyclophosphamide (46% ever, 20% current), and lenalidomide (28% ever, 15% current). Many patients reported combination treatments, including cyclophosphamide+bortezomib+dexamethasome (CyBorD, 17% current). Half of the patients (51%) had received three or more different treatments. Nearly three-quarters (71%, n=226) reported ever having problems tolerating AL amyloidosis treatment, of which nearly half (47%, n=107) had discontinued at least one treatment. Nearly half (46%) of those currently being treated reported some tolerability issue (less than very good tolerability). Tolerability varied across the common treatments from a low of 3.22 (SD=0.90) for cyclophosphamide to a high of 3.61 (SD=0.52) for SCT. Problems with tolerating current medications corresponded with decrements in HRQoL (both MCS and PCS, p<0.001).
Conclusion
Lifetime history of TRSs was high. Discontinuation of life-saving AL amyloidosis treatments was fairly common, though most patients were able to tolerate their current regimen. The high prevalence of treatment discontinuation and history of multiple AL amyloidosis treatments suggests that physicians and patients try a variety of treatments to balance tolerability and efficacy. TRSs are associated with decrements in HRQoL, over and above the burden of AL amyloidosis. These findings highlight the importance of assessing HRQoL during treatment for AL amyloidosis to better understand tolerability, and the need for more treatment options for AL amyloidosis, particularly those with favorable tolerability. Study supported by: Prothena Biosciences Inc
Session topic: E-poster
Keyword(s): AL amyloidosis, Quality of life, Tolerance
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