What the trial was testing
The APOLLO-B enrolled 360 patients with amyloidosis. The study was sponsored by Alnylam Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Patisiran helped preserve walking ability and improved quality of life scores in people with ATTR heart disease.
The New England journal of medicine · 2023 · NCT03997383
These findings — that people on patisiran maintained their ability to walk farther than those on placebo after one year. — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 360 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with amyloidosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Patisiran is FDA-approved for ATTR amyloidosis with nerve damage, and this trial shows it may help people with the heart form of the disease. If you have ATTR heart disease, ask your cardiologist whether patisiran is right for you and whether it's covered by your insurance.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open amyloidosis trials
A Prospective Cohort Study on Primary Cutaneous Amyloidosis
The aim of this clinical trial is to find out whether the combination of tocilizumab tablets and acitretin capsules is more effective than acitretin capsules alone in treating primary cutaneous amyloidosis. It will also investigate the safety of the combination of tocilizumab tablets and acitretin capsules. The main questions it aims to answer are: 1. Does the combination of tocilizumab tablets and acitretin capsules relieve the pruritus symptoms of the participants faster and reduce the pruritus score more than acitretin capsules alone? 2. What medical problems will the participants encounter when taking tocilizumab tablets combined with acitretin capsules? The researchers compared the combination of tocilizumab tablets and acitretin capsules with acitretin capsules alone to see if the combination could better treat primary cutaneous amyloidosis without causing serious adverse reactions. Participants will: 1. Take the combination of tocilizumab tablets and acitretin capsules or acitretin capsules alone every day for 16 weeks. 2. Visit the clinic once every 4 weeks for checkups and tests. 3. Observe participants at 4 weeks, 12 weeks and 24 weeks after discontinuation of medication to determine the recurrence status. 4. Collect the visual analogue scale (VAS) scores for pruritus, symptom severity (SCORAD) scores , rash area and severity, treatment response (EASI) scores, dermatological quality of life index (DLQI), and insomnia severity index (ISI) of participants before and after treatment.
Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases (EchoNet-Screening)
Despite rapidly advancing developments in targeted therapeutics and genetic sequencing, persistent limits in the accuracy and throughput of clinical phenotyping has led to a widening gap between the potential and the actual benefits realized by precision medicine. Recent advances in machine learning and image processing techniques have shown that machine learning models can identify features unrecognized by human experts and more precisely/accurately assess common measurements made in clinical practice. The investigators have developed an algorithm, termed EchoNet-LVH, to identify cardiac hypertrophy and identify patients who would benefit from additional screening for cardiac amyloidosis and will prospectively evaluate its accuracy in identifying patients whom would benefit from additional screening for cardiac amyloidosis.