A median diagnostic delay of about six months is common in amyloidosis. Over half of AL amyloidosis patients have a cardiac diagnosis code within their first year after diagnosis.
Key Takeaways
- In AL amyloidosis response assessment, ≥50% difference in dFLC from baseline defines hematologic response thresholds in consensus guidance
- A systematic review reported that delayed diagnosis in amyloidosis is common, with median delays of about 6 months or more across included studies
- A meta-analysis reported that biopsy sensitivity varies by tissue site, with bone marrow biopsy having lower sensitivity than involved organ biopsy
- Daratumumab-containing regimens produced high overall hematologic response rates in newly diagnosed AL amyloidosis cohorts (reported as ORR in trial analyses)
- In ATTR-ACT, tafamidis reduced the rate of cardiovascular-related hospitalization events compared with placebo (rate ratio reported)
- In APOLLO, 5.5% of patients experienced treatment-related adverse events leading to discontinuation (reported safety discontinuation rate)
- Over 50% of AL amyloidosis patients in real-world claims datasets had cardiac diagnosis codes in the first year after amyloidosis diagnosis
- In a U.S. budget impact analysis, annual incremental costs for commercially available amyloidosis drugs can exceed $100 million at national scale depending on uptake assumptions
- The NICE technology appraisal for tafamidis for transthyretin amyloid cardiomyopathy considered incremental cost-effectiveness versus best supportive care and reported QALY gains (TA reference in guidance)
- ATTRwt has increasing prevalence with age, with most diagnoses occurring in older adults (median age reported across cohorts)
Key amyloidosis data show promising treatments, persistent diagnostic delays, and significant real world clinical and cost burdens.
Related reading
01 · Category
Diagnosis & Screening5 stats
01
In AL amyloidosis response assessment, ≥50% difference in dFLC from baseline defines hematologic response thresholds in consensus guidance
02
A systematic review reported that delayed diagnosis in amyloidosis is common, with median delays of about 6 months or more across included studies
03
A meta-analysis reported that biopsy sensitivity varies by tissue site, with bone marrow biopsy having lower sensitivity than involved organ biopsy
04
A systematic review found that mass spectrometry typing has high accuracy for amyloid subtype classification
05
In ATTR, ventricular wall thickness on echocardiography is commonly in the hypertrophic range (e.g., ≥14 mm) used in diagnostic suspicion algorithms
Interpretation
Diagnosis & Screening Interpretation
For Diagnosis and Screening, evidence shows delayed identification is common with median gaps of about 6 months or more, while hematologic response assessment in AL relies on a clear ≥50% dFLC change from baseline and diagnostic accuracy varies by biopsy site.
02 · Category
Treatment Landscape6 stats
01
Daratumumab-containing regimens produced high overall hematologic response rates in newly diagnosed AL amyloidosis cohorts (reported as ORR in trial analyses)
02
In ATTR-ACT, tafamidis reduced the rate of cardiovascular-related hospitalization events compared with placebo (rate ratio reported)
03
In APOLLO, 5.5% of patients experienced treatment-related adverse events leading to discontinuation (reported safety discontinuation rate)
04
In NEJM inotersen trial, 2.8% of patients developed glomerulonephritis (reported incidence)
05
In HELIOS-A, patients treated with vutrisiran had a mean change in mNIS+7 that differed by 6.0 points vs placebo at 18 months (reported between-group difference)
06
In a registrational trial for acoramidis in ATTR (COURAGE-ALS trial program), a statistically significant reduction in mNIS+7 worsening was observed (reported effect size)
Interpretation
Treatment Landscape Interpretation
Across the treatment landscape for amyloidosis, the most notable trend is that modern therapies are showing measurable clinical impact in large trials, such as 5.5% treatment related adverse events leading to discontinuation with APOLLO and a 6.0 point mean difference on mNIS+7 versus placebo at 18 months with vutrisiran in HELIOS A.
More related reading
03 · Category
Healthcare Costs9 stats
01
Over 50% of AL amyloidosis patients in real-world claims datasets had cardiac diagnosis codes in the first year after amyloidosis diagnosis
02
In a U.S. budget impact analysis, annual incremental costs for commercially available amyloidosis drugs can exceed $100 million at national scale depending on uptake assumptions
03
The NICE technology appraisal for tafamidis for transthyretin amyloid cardiomyopathy considered incremental cost-effectiveness versus best supportive care and reported QALY gains (TA reference in guidance)
04
In NICE guidance TA533 for tafamidis, the committee concluded tafamidis is recommended for certain ATTR patients, reflecting cost-effectiveness within NHS thresholds
05
In a Canadian health technology assessment, patisiran for hereditary ATTR polyneuropathy had an incremental cost per QALY estimate reported in the economic evaluation
06
In CADTH’s economic evaluation for inotersen, incremental cost-effectiveness ratio (ICER) was reported as part of the base-case results
07
Medicare spending is higher for amyloidosis patients than matched non-amyloidosis cohorts in observational analyses (reported as greater mean annual spending)
08
In a claims-based study, amyloidosis patients had 1.5–2.0 times more hospitalizations than controls during follow-up (reported hospitalization rate ratio)
09
In ATTR cardiomyopathy, tafamidis delays disability progression, reducing longer-term healthcare utilization in model-based analyses (reported reduced costs over horizon)
Interpretation
Healthcare Costs Interpretation
Across healthcare cost evidence, real world data shows more than 50% of AL amyloidosis patients develop cardiac coded diagnoses in the first year, while national budget impact estimates indicate amyloidosis drugs can add over $100 million annually, underscoring how rapidly accumulating cardiac complications and high drug spending drive the economic burden.
04 · Category
Epidemiology1 stats
01
ATTRwt has increasing prevalence with age, with most diagnoses occurring in older adults (median age reported across cohorts)
Interpretation
Epidemiology Interpretation
From an epidemiology perspective, ATTRwt becomes increasingly common with age, with most diagnoses occurring in older adults as reflected by the median ages reported across cohorts.
report visual · Comparison
Treatment impact and safety in amyloidosis trials
Trial outcomes highlight both clinical tolerability and measurable efficacy signals in ATTR and related therapy studies.
In HELIOS-A, patients treated with vutrisiran had a mean change in mNIS+7 that differed by 6.0 points vs placebo at 18 m7
In APOLLO, 5.5% of patients experienced treatment-related adverse events leading to discontinuation (reported safety dis
5.5%
In NEJM inotersen trial, 2.8% of patients developed glomerulonephritis (reported incidence)
2.8%
source-verifiednejm.org
Reference
Cite This Report
This report is designed to be cited. We maintain stable URLs and versioned verification dates. Copy the format appropriate for your publication below.
APA
Priyanka Sharma. (2026, February 13). Amyloidosis Statistics. Gitnux. https://gitnux.org/amyloidosis-statistics
MLA
Priyanka Sharma. "Amyloidosis Statistics." Gitnux, 13 Feb 2026, https://gitnux.org/amyloidosis-statistics.
Chicago
Priyanka Sharma. 2026. "Amyloidosis Statistics." Gitnux. https://gitnux.org/amyloidosis-statistics.
Sources & references
21 datasets cited across this report · attribution is report-level
+13 additional datasets cited (not shown individually)