PNH Clinical Trial & Hematology Evidence Updates Ledger
Unlike a generic blog, this ledger records only verified clinical milestones, Phase 3 hematology trial readouts, and archival stewardship dispatches. Every entry is audited against primary publications in The New England Journal of Medicine, Blood, and The Lancet Haematology.
This chronological ledger tracks regulatory approvals, long-term Phase 3 extension trials, eculizumab biosimilar launches, and guideline updates in Paroxysmal Nocturnal Hemoglobinuria (PNH). Every entry specifies its verification date, primary journal DOI, and how the evidence changes practical decisions for patients and hematologists.
| Audit Date & Type | Dispatch / Trial Title | Key Clinical Takeaway & Verification Source | Dossier Link |
|---|---|---|---|
| 2026-11-09 MANIFESTO |
From Biweekly IV Tubes to a Morning Capsule: Why We Rebuilt an 18-Year PNH Domain | Full 2003-2026 stewardship manifesto detailing the transition from the pre-Soliris era to oral Factor B (iptacopan), subcutaneous C3 (pegcetacoplan), Factor D add-on (danicopan), and recycling C5 antibodies. | Read Full Dispatch -> |
| 2026-10-15 TRIAL AUDIT |
APPLY-PNH & APPOINT-PNH 48-Week Long-Term Oral Iptacopan Extension | Confirmed durable hemoglobin normalization (>=12 g/dL in ~69% of patients), sustained transfusion avoidance (>92%), and low clinical breakthrough hemolysis rates with twice-daily oral Factor B inhibition (NEJM / ASH Follow-up). | Complement Matrix -> |
| 2026-08-20 DUAL BLOCKADE |
ALPHA Trial & FDA Approval of Oral Danicopan (Voydeya) Add-On for EVH | Demonstrated that adding oral Factor D inhibition (danicopan TID) onto background ravulizumab or eculizumab raises hemoglobin by ~+2.9 g/dL while preserving terminal C5 protection against severe BTH (Lee et al., Lancet Haematol). | Complement Matrix -> |
| 2026-06-12 GLOBAL ACCESS |
Subcutaneous Crovalimab (Piasky) & Eculizumab Biosimilars (Bkemv, Epysqli) | Tracked FDA/EMA/NMPA approvals of 4-week subcutaneous crovalimab (effective in C5 Arg885His carriers) and interchangeable eculizumab biosimilars expanding global affordability. | Global Access -> |
Frequently Asked Hematology & Patient Questions
Direct, link-free clinical answers grounded in ASH, IPIG, and Phase 3 complement inhibitor trial evidence.
How does the Evidence Ledger select which PNH studies to feature?
We prioritize randomized Phase 3 clinical trials, multi-year international PNH registry analyses, consensus flow cytometry standards, and official health technology reimbursement rulings that directly impact patient survival, hemoglobin normalization, thrombotic safety, or financial access.
Methodology: See About & Evidence Methodology.