High-Dose Melphalan and Autologous Peripheral Blood Stem Cell Transplantation in AL Amyloidosis

Sanchorawala, Vaishali

doi:10.1159/000506498

Cited by 20 publications

(15 citation statements)

References 46 publications

(47 reference statements)

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“…Despite an evolving therapeutic landscape for AL amyloidosis, HDM/SCT remains one of the most effective treatments available 40,41 . In our study, racial/ethnic minorities were less likely to receive treatment with HDM/SCT, with Hispanics in particular having a 30% lower likelihood compared to NHWs.…”

Section: Discussionmentioning

confidence: 99%

Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities

et al. 2020

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In marked contrast to multiple myeloma, racial/ethnic minorities are underrepresented in publications of systemic light-chain (AL) amyloidosis. The impact of race/ethnicity is therefore lacking in the narrative of this disease. To address this gap, we compared disease characteristics, treatments, and outcomes across racial/ethnic groups in a referred cohort of patients with AL amyloidosis from 1990 to 2020. Among 2416 patients, 14% were minorities. Non-Hispanic Blacks (NHBs) comprised 8% and had higher-risk sociodemographic factors. Hispanics comprised 4% and presented with disproportionately more BU stage IIIb cardiac involvement (27% vs. 4–17%). At onset, minority groups were younger in age by 4–6 years. There was indication of more aggressive disease phenotype among NHBs with higher prevalence of difference between involved and uninvolved free light chains >180 mg/L (39% vs. 22–33%, P = 0.044). Receipt of stem cell transplantation was 30% lower in Hispanics compared to non-Hispanic White (NHWs) on account of sociodemographic and physiologic factors. Although the age/sex-adjusted hazard for death among NHBs was 24% higher relative to NHWs (P = 0.020), race/ethnicity itself did not impact survival after controlling for disease severity and treatment variables. These findings highlight the complexities of racial/ethnic disparities in AL amyloidosis. Directed efforts by providers and advocacy groups are needed to expand access to testing and effective treatments within underprivileged communities.

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Section: Discussionmentioning

confidence: 99%

Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities

et al. 2020

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“…The first stage in the management of toxicity in relation to ASCT is during stem cell mobilization and collection whereby low serum albumin, elevated NT-proBNP and increased septal thickening were found to be important risk factors for toxicity 34 , 35 During stem cell mobilization, patients can experience various toxicities including tachyarrhythmias, thromboembolic events, weight gain (due to fluid retention), bleeding, acute kidney injury, hypertensive crises, or hypotension. To minimize risk of toxicity, it is recommended to use granulocyte-colony-stimulating factor (G-CSF) without cyclophosphamide given the increased cardiac morbidity, significantly higher number of apheresis required, increased hospitalizations and increased toxicity associated with the latter 36 . The recommended dose of G-CSF is 10–16 μg/kg/day, either as one dose or divided into two doses, 3 days before stem cell collection for an optimal total of at least 5 × 10 6 CD34+ cells/kg 37 .…”

Section: Treatment Of Asct-eligible Patientsmentioning

confidence: 99%

“…The general recommendation though, is to split the dose. If patients fail stem cell mobilization, plerixafor is a well-tolerated adjuvant 36 .…”

Section: Treatment Of Asct-eligible Patientsmentioning

confidence: 99%

Comprehensive Review of AL amyloidosis: some practical recommendations

Hamed

Bazarbachi

et al. 2021

Blood Cancer J.

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Amyloid light chain (AL) amyloidosis is among the more common and more severe of the amyloidoses usually involving the slow proliferation of a bone-marrow-residing plasma cell (PC) clone and the secretion of unstable immunoglobulin-free light chains (FLC) that infiltrate peripheral tissues and result in detrimental end-organ damage. Disease presentation is rather vague, and the hallmark of treatment is early diagnosis before irreversible end-organ damage. Once diagnosed, treatment decision is transplant-driven whereby ~20% of patients are eligible for autologous stem cell transplantation (ASCT) with or without bortezomib-based induction. In the setting of ASCT-ineligibility, bortezomib plays a central role in upfront treatment with the recent addition of daratumumab to the current emerging standard of care. In general, management of AL amyloidosis is aimed at achieving deep, durable responses with very close monitoring for early detection of relapse/refractory disease. This article provides a comprehensive review of the management of patients with AL amyloidosis including goals of therapy, current treatment guidelines in the setting of both ASCT-eligibility and ineligibility, treatment response monitoring recommendations, toxicity management, and treatment of relapse/refractory disease.

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“…High-dose intravenous melphalan and autologous SCT was developed for the treatment of AL amyloidosis in the early 1990s and was prompted by its success in myeloma. 3 This application has evolved significantly over the past 3 decades. These guidelines provide a comprehensive assessment of eligibility criteria, stem cell collection and mobilization strategies and regimens, risk-adapted melphalan dosing, role for induction and consolidation therapies, specific supportive care management, long-term outcome with respect to survival, hematologic response and relapse, and organ responses following SCT.…”

mentioning

confidence: 99%

High-Dose Melphalan and Autologous Peripheral Blood Stem Cell Transplantation in AL Amyloidosis

Cited by 20 publications

References 46 publications

Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities

Race/ethnicity in systemic AL amyloidosis: perspectives on disease and outcome disparities

Comprehensive Review of AL amyloidosis: some practical recommendations

Summary of the EHA-ISA Working Group Guidelines for High-dose Chemotherapy and Stem Cell Transplantation for Systemic AL Amyloidosis

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