A longitudinal evaluation of health‐related quality of life in patients with <scp>AL</scp> amyloidosis: associations with health outcomes over time

Sanchorawala, Vaishali; McCausland, Kristen L.; White, Michelle; Bayliss, Martha; Guthrie, Spencer; Lo, Stephen; Skinner, Martha

doi:10.1111/bjh.14889

Cited by 28 publications

(35 citation statements)

References 14 publications

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“…Our results show that baseline PROs can be helpful in predicting 1-year (early) mortality in AL amyloidosis even after adjusting for stage at diagnosis. The Boston University Amyloid Center has previously shown health-related quality of life to be of prognostic value with worse pre-treatment physical component scores on the SF-36 associated with a greater risk of mortality in patients who received transplant or non-transplant chemotherapy 20 , 21 . These analyses have shown that the pre-treatment physical component score of the SF-36 can predict post-treatment mortality after adjusting for the number of organs affected 21 .…”

Section: Discussionmentioning

confidence: 99%

“…The Boston University Amyloid Center has previously shown health-related quality of life to be of prognostic value with worse pre-treatment physical component scores on the SF-36 associated with a greater risk of mortality in patients who received transplant or non-transplant chemotherapy 20 , 21 . These analyses have shown that the pre-treatment physical component score of the SF-36 can predict post-treatment mortality after adjusting for the number of organs affected 21 . Similarly, the Mayo Clinic has shown that a 3-question screen of patient-reported fatigue, pain, and quality of life can be useful in AL amyloidosis with fatigue independently predicting survival in addition to stage and transplant status 22 .…”

Section: Discussionmentioning

confidence: 99%

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Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

et al. 2021

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We conducted a prospective cohort study in newly diagnosed systemic light chain (AL) amyloidosis patients (N = 59) to study patient-reported outcomes (PROs) through the first year. The median age was 68 years with 42% female, 8% Black, and 78% lambda subtype. Organ involvement was cardiac in 66%, renal in 58%, with 25% having 3 or greater organs involved. Between baseline and 3 months, all PROMIS®-29 domain scores worsened by 0.4–4.1 points except anxiety which improved by 2.1 points. By 1 year, scores improved compared to the greatest decline at 3 months, most statistically significant for global physical health, physical function, and fatigue. On stage-adjusted survival analysis, in addition to baseline global physical and mental health, domains measuring physical function, fatigue, anxiety, depression, and social roles were associated with 1-year survival. At 1 year, PROMIS measures were associated with NT-proBNP changes and hematologic response. Among patients with an NT-proBNP response, the improvement was seen in physical function, social roles, global mental health, and anxiety. Among patients with an NT-proBNP progression, worsening was seen with anxiety, depression, sleep, and global mental health. Measuring and tracking PROs in patients with AL amyloidosis is important and these important outcomes can be used as correlative endpoints in clinical care/research.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

et al. 2021

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“…Collaborative symptom management with rehabilitation physicians and/or palliative medicine teams might also be of value. In one observational study 176 , significant improvements in vitality, social functioning, role-emotional and mental health were demonstrated after HDM/SCT in patients with AL amyloidosis 176 . Lower pre-treatment SF-36 physical component scores were associated with a greater risk of mortality in patients receiving HDM/SCT or those who received non-SCT chemotherapy regiments and during follow-up periods 176 .…”

Section: [H2] Amyloid Directed Immunotherapymentioning

confidence: 99%

“…In one observational study 176 , significant improvements in vitality, social functioning, role-emotional and mental health were demonstrated after HDM/SCT in patients with AL amyloidosis 176 . Lower pre-treatment SF-36 physical component scores were associated with a greater risk of mortality in patients receiving HDM/SCT or those who received non-SCT chemotherapy regiments and during follow-up periods 176 . An improvement in SF-36 scores after HDM/SCT was also demonstrated in another observational study 99 ; mental component summary scores reached the population norm 1 year post-SCT and physical component summary score reached the population norm 2 years post-SCT.…”

Section: [H2] Amyloid Directed Immunotherapymentioning

confidence: 99%

Systemic immunoglobulin light chain amyloidosis

Merlini

Dispenzieri

Sanchorawala

et al. 2018

Nat Rev Dis Primers

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407

387

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Systemic immunoglobulin light chain (AL) amyloidosis is a protein misfolding disease that is caused by the conversion of immunoglobulin light chains from their soluble functional states into highly organized amyloid fibrillar aggregates that lead to organ dysfunction. The disease is progressive and, accordingly, early diagnosis is vital to prevent irreversible organ damage, of which cardiac and renal damage predominate. The development of novel sensitive biomarkers and imaging technologies for the detection and quantification of organ involvement and damage are facilitating earlier diagnosis and enhanced evaluation of the efficacy of new and existing therapies. Treatment is guided by risk assessment which is based on levels of cardiac biomarkers; close monitoring of clonal and organ response guides duration of therapy and changes in regimen. Several new classes of drugs, such as proteasome inhibitors and immunomodulatory drugs, along with high-dose chemotherapy and autologous haematopoietic stem cell transplantation, have led to rapid and deep suppression of the amyloid light chain production in the majority of patients. However, effective therapies for patients with advanced cardiac involvement are an unmet need. Passive immunotherapies targeting clonal plasma cells and directly accelerating removal of amyloid deposits promise to further improve the overall outlook of this increasingly treatable disease.

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“…Following treatment, SF-36 scores improved; MCS reached the population norm 1-year post HDM-SCT and PCS reached the population norm 2 years post HDM-SCT [34]. Levels of cardiac biomarkers (NT-proBNP) correlated with mean SF-36 score; an association between risk of death and baseline SF-36 PCS scores has also been shown [35]. SF-36 scores were informative when examining factors associated with early post-treatment survival and subsequent survival beyond 1-year follow-up.…”

Section: Burden Of Illness: Detecting Clinically Meaningful Impacts Omentioning

confidence: 87%

The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis

Lousada

2020

Orphanet J Rare Dis

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Background Immunoglobulin light chain (AL) amyloidosis is a rare, multi-systemic disorder characterized by two disease processes: an underlying plasma cell dyscrasia that provides the source of pathologic light chains, and the resulting organ dysfunction caused by deposition of amyloid light chain fibrils. There are no FDA approved treatments for AL amyloidosis; regimens developed for multiple myeloma are used off-label to treat the plasma cell disorder and no therapies are directed at organ deposition. Thus, an unmet medical need persists despite advances in disease management. A public-private partnership was recently formed between the Amyloidosis Research Consortium (ARC) and the US Food and Drug Administration (FDA) to bridge scientific gaps in drug development for the treatment of AL amyloidosis. Main Body The inaugural Amyloidosis Forum was convened at FDA on 12 November 2019 and led by a multidisciplinary panel of physicians, health outcomes professionals, and representatives from the FDA, ARC, and pharmaceutical companies. Patients provided important perspectives on the pathway to diagnosis, challenges of rigorous treatment, and the burden of disease. The panel reviewed the epidemiology, pathobiology, and clinical features of AL amyloidosis. Hematologic characteristics, staging systems, and response criteria were examined with clear consensus that a “deep response” to plasma cell-directed treatments was critical to overall survival. Emphasis was placed on the heterogeneous clinical phenotypes of AL amyloidosis, including cardiovascular, renal, neurological, and gastrointestinal system manifestations that contribute to morbidity and/or mortality, but render challenges to clinical trial endpoint selection. FDA representatives discussed regulatory perspectives regarding demonstration of clinical benefits of investigational therapies in the context of a rare disease with multi-systemic manifestations. The panel also highlighted the potential importance of well-designed health-related quality of life instruments, quantification of system organ effects, the potential of advanced imaging technologies, and survival prediction models. Conclusions The Amyloidosis Forum identified a clear need for novel trial designs that are scientifically rigorous, feasible, and incorporate clinically meaningful endpoints based on an understanding of the natural history of the disease in an evolving therapeutic landscape. Future forums will delve into these issues and seek to include participation from additional stakeholders.

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A longitudinal evaluation of health‐related quality of life in patients with AL amyloidosis: associations with health outcomes over time

Cited by 28 publications

References 14 publications

Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

Changes in patient-reported outcomes in light chain amyloidosis in the first year after diagnosis and relationship to NT-proBNP change

Systemic immunoglobulin light chain amyloidosis

The Amyloidosis Forum: a public private partnership to advance drug development in AL amyloidosis

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