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Guidelines for non-transplant chemotherapy for treatment of systemic AL amyloidosis: EHA-ISA working group
Ashutosh D Wechalekar1, M Teresa Cibeira2, Simon D Gibbs3
1National Amyloidosis Centre, University College London (Royal Free Campus), London, UK.
Insights
This guideline recommends non-transplant chemotherapy for AL amyloidosis, prioritizing treatment based on patient presentation and tolerance. It suggests daratumumab-VCD for untreated patients and individualized relapse strategies.
Area of Science:
- Hematology
- Oncology
- Internal Medicine
Background:
- Guidelines for AL amyloidosis treatment developed by the European Society of Haematology (ESH) and International Society of Amyloidosis (ISA).
- Focuses on non-transplant chemotherapy strategies for AL amyloidosis patients.
Framework:
- Recommendations based on literature review, evidence grading, and expert consensus from ESH and ISA committees.
- Treatment selection guided by clinical presentation, patient tolerance, and potential side effects.
Implementation:
- Recommends daratumumab-VCD for most untreated AL amyloidosis patients; VCD or VMDex if daratumumab is unavailable.
- Relapse treatment considers initial response depth/duration, prior agent exposure, patient fitness, and organ damage.
- Encourages AL amyloidosis patient participation in available clinical trials.
Implications:
- Highlights the need for prospective evaluation of targeted agents like venetoclax.
- Stresses the importance of including advanced-stage patients in future trials for evidence-based decisions.
- Calls for novel therapies targeting amyloid fibrils or reducing proteotoxicity of light chains.
Background:
This guideline has been developed jointly by the European Society of Haematology and International Society of Amyloidosis recommending non-transplant chemotherapy treatment for patients with AL amyloidosis.
Methods:
A review of literature and grading of evidence as well as expert recommendations by the ESH and ISA guideline committees.
Results And Conclusions:
The recommendations of this committee suggest that treatment follows the clinical presentation which determines treatment tolerance tempered by potential side effects to select and modify use of drugs in AL amyloidosis. All patients with AL amyloidosis should be considered for clinical trials where available. Daratumumab-VCD is recommended from most untreated patients (VCD or VMDex if daratumumab is unavailable). At relapse, the two guiding principles are the depth and duration of initial response, use of a class of agents not previously exposed as well as the limitation imposed by patients' fitness/frailty and end organ damage. Targeted agents like venetoclax need urgent prospective evaluation. Future prospective trials should include advanced stage patients to allow for evidence-based treatment decisions. Therapies targeting amyloid fibrils or those reducing the proteotoxicity of amyloidogenic light chains/oligomers are urgently needed.
