Amyloidosis — Research Summary
Printed from RareWays (rareways.com.au) on 5 April 2026
For general awareness only. Not medical advice. Discuss all care options with your healthcare team.
5 Most Recent Research Articles
- 1.
Frequency-adjusted daratumumab-based regimen versus bortezomib/dexamethasone in newly diagnosed AL amyloidosis: a matched-cohort study.
Zheng Wanting et al. — Annals of medicine (1 December 2026)
https://pubmed.ncbi.nlm.nih.gov/41560662/
- 2.
Hidden overlap between heart failure with preserved ejection fraction and transthyretin amyloid cardiomyopathy: Why it matters and how to avoid missing it.
Tanaka Hidekazu — International journal of cardiology (1 May 2026)
https://pubmed.ncbi.nlm.nih.gov/41698549/
- 3.
Beyond the valve: The long shadow of transthyretin amyloidosis in aortic stenosis.
Cantone Anna et al. — International journal of cardiology (15 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41621489/
- 4.
Cardiac remodeling and arterial stiffness progression in wild-type vs hereditary transthyretin amyloidosis in Crete.
Korela Dafni et al. — International journal of cardiology (15 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41525938/
- 5.
Ultrasonication-induced in vitro formation of transthyretin mature amyloid fibrils at neutral pH.
Yuzu Keisuke et al. — Protein science : a publication of the Protein Society (1 April 2026)
https://pubmed.ncbi.nlm.nih.gov/41793160/
Clinical Trials — Currently Recruiting (Australia)
Ask your doctor whether you or your child may be eligible for any of these trials.
- 1.
Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients
Recruiting — Phase 1 — Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
https://clinicaltrials.gov/study/NCT06395285
- 2.
TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
Recruiting — Phase 3 — Alnylam Pharmaceuticals
https://clinicaltrials.gov/study/NCT07052903
- 3.
A Study of a Potential Disease Modifying Treatment in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation
Recruiting — Phase 2 — Washington University School of Medicine
https://clinicaltrials.gov/study/NCT06647498
- 4.
A Study to Assess Change in Disease Activity and Adverse Events (AE)s in Adult Participants With Immunoglobulin Light Chain (AL) Amyloidosis Receiving Etentamig (ABBV-383) as an Intravenous (IV) Infusion
Recruiting — Phase 1 — AbbVie
https://clinicaltrials.gov/study/NCT06158854
- 5.
Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant
Recruiting — Phase 3 — Eidos Therapeutics, a BridgeBio company
https://clinicaltrials.gov/study/NCT06563895
- 6.
A Phase II Trial of Teclistamab in Participants With Previously Treated Immunoglobulin Light-chain (AL) Amyloidosis
Recruiting — Phase 2 — European Myeloma Network B.V.
https://clinicaltrials.gov/study/NCT06649695
- 7.
Study of AT-02 in Healthy Volunteers and Subjects With Systemic Amyloidosis
Recruiting — Phase 1 — Attralus, Inc.
https://clinicaltrials.gov/study/NCT05521022
- 8.
Combined Dose-Finding and CV Outcomes Study With CSL300 (Clazakizumab) in Adult Subjects With ESKD Undergoing Dialysis
Recruiting — Phase 2 — CSL Behring
https://clinicaltrials.gov/study/NCT05485961
- 9.
A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation
Recruiting — Phase 2 — Washington University School of Medicine
https://clinicaltrials.gov/study/NCT05552157
- 10.
CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) Amyloidosis
Recruiting — Phase 3 — Novo Nordisk A/S
https://clinicaltrials.gov/study/NCT07207811
Source: RareWays research directory. Data from PubMed, Europe PMC, OpenAlex, ClinicalTrials.gov.
Always verify information with your healthcare team before making any decisions about your care.
Amyloidosis
Amyloidosis is a group of conditions where abnormal proteins called amyloid fibres build up in organs, causing progressive organ damage. ATTR amyloidosis (transthyretin) and AL amyloidosis are the most common types. New RNA-based therapies and small molecule stabilisers have significantly improved outcomes.
Most Recent Research
BACKGROUND: The optimal dosing schedule for daratumumab (Dara) in newly diagnosed systemic light-chain (AL) amyloidosis requires refinement. This real-world study compared the efficacy and safety of a frequency-adjusted, cyclophosphamide-free Dara-based regimen with bortezomib/dexamethasone (BD). METHODS: We included newly diagnosed AL amyloidosis patients treated between January 2018 and December 2024, with follow-up data censored in August 2025. Using 1:1 propensity score matching based on age, cardiac stage, dFLC, and organ function, we compared hematologic/organ responses and survival. Survival was analyzed with Kaplan-Meier and log-rank tests. The Dara-based group received a cyclophosphamide-free regimen with an adjusted schedule (biweekly in cycle 1, then monthly) and a response-guided treatment duration. RESULTS: A total of 105 patients were included. After propensity score matching, 60 patients (30 per group) were selected for comparative analysis. The Dara-based group achieved significantly higher hematologic complete response (CR) rates at 6 months (57% vs. 27%, p = 0.018) and 12 months (63% vs. 27%, p = 0.002), with a markedly shorter median time to CR (61 vs. 120 days, p = 0.010). Organ responses were also superior: cardiac response 52% vs. 24% (p = 0.037) and renal response 56% vs. 27% (p = 0.037). No significant survival difference was observed during follow-up, and the safety profile was comparable between groups. CONCLUSION: A frequency-adjusted Dara-based regimen induces significantly faster and deeper hematologic and organ responses compared to BD in newly diagnosed AL amyloidosis which offers a promising personalized approach to reduce treatment burden and adverse events while maintaining high efficacy, supporting its integration into clinical practice for a broader patient population.
This information is for general awareness only.
For guidance specific to your situation, please speak with your healthcare team.