Background: Immunoglobulin light chain (AL) amyloidosis is a plasma cell neoplasm characterized by deposition of intrinsically unstable free light chain (FLC) fibrils in vital organs, such as the heart and kidneys. AL amyloidosis can be rapidly fatal in patients with advanced cardiac involvement and symptomatology can have profound impacts on quality of life. AL amyloidosis is not curable. Available therapies target the underlying plasma cell clone to halt amyloid production and arrest progressive organ dysfunction, but do not directly impact amyloid fibril removal. Despite improved outcomes with daratumumab and bortezomib-based regimens, early mortality and poor quality of life remain clinically unmet needs. High symptom burden, multiorgan involvement, and risk of early death underscore the need for multidisciplinary care teams. We hypothesize that early integration of palliative care (PC) may be beneficial in addressing symptoms, establishing goals of care, and overall, holistically alleviating suffering of AL amyloidosis patients.

Methods: We retrospectively reviewed 19 AL amyloidosis patients referred to PC between January 2020-March 2024 at DFCI/BWH Amyloidosis Program. Each patient received at least one ambulatory and/or inpatient PC consultation. Baseline demographics, treatment, and PC interventions were extracted from medical records. PC delivery was characterized utilizing the National Coalition for Hospice and Palliative Care guidelines. The 2004 Mayo Clinic staging with European modifications was used for cardiac staging. Hematological and organ responses were assessed according to the International Society of Amyloidosis consensus criteria.

Results: Median age was 69 years (range 39-84) and eight (42.1%) were female. Sixty-eight percent were newly diagnosed and 31.6% relapsed after a median of two lines of therapy (range 1-3). One patient had concurrent multiple myeloma. Sixteen (84%) had advanced cardiac involvement (equally split between Mayo stage IIIA and stage IIIB), and eleven (57.9%) had renal involvement, including two on renal replacement therapy (RRT) at time of referral. Median number of organs involved was three (range 1-6). Nearly all patients (95%) received frontline daratumumab-based therapy. Overall hematologic response after first-line treatment was 95%. All patients (100%) achieved a VGPR or better with 79% achieving a CR. Cardiac responses included 5% carCR, 32% carVGPR, and 26% cardiac PR, with median time to best cardiac response of 14.5 months (range 5-42). Median time from diagnosis to PC referral was 84 days (range 10-1233). Referral reasons included symptom management (47%), goals of care (32%), cardiac transplant evaluation (16%), and suitability for RRT (5%). At 30-month median follow-up, patients had a median of five hospital/ED visits (range 1-22) or 3.6/year (range 0.6-8.4). Fourteen patients (73.7%) remain alive and in ≥VGPR, while five (26.3%) died from progressive amyloidosis (median OS from diagnosis: 25 months, range 4-54 months; median OS from PC referral: 74 days, range 20-167). Patients had an average of 10 PC visits (62.6% inpatient) and seen a median of every 24.7 days (range 1-675). Common PC interventions included symptom assessment (100% patients), screening emotional distress/maladaptive coping (94.7%), goals of care (84.2%), psychosocial evaluation (89.5%), treatment of spiritual distress (31.6%), and end-of-life care coordination (26.3%). Top symptoms per patient were pain (17%), anorexia (16%), sleep (15%), GI/nausea (14%), fatigue (13%), and dyspnea (13%). Pain was the most frequently documented symptom (33% notes).

Conclusion: Despite high rates of hematologic response, AL amyloidosis patients are gravely affected by significant symptom burden due to delayed/absent organ recovery and/or treatment-related toxicities. Furthermore, AL amyloidosis patients and their families face critical decisions early in their disease course regarding utilization of advanced therapies, such as RRT or organ transplantation. PC interventions address a broad spectrum of needs—including physical, emotional, and psychosocial support—but referrals often occurred late in the disease course. Our preliminary data support the role for early integration of PC in the care of AL amyloidosis patients to improve quality of life, set goals of care, and reduce healthcare utilization. Prospective studies are warranted to evaluate structured, early PC models in AL amyloidosis.

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2025
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