| Evidence area | Study / source | Key quantitative or translational findings | URL | Publication / registry date | Citation |
|---|---|---|---|---|---|
| ALPS genetic testing cohort | Xu et al., *Journal of Clinical Immunology*; retrospective ALPS NGS testing cohort | 802 patients tested (May 2014–Jan 2023); median age 12 years; 63% male (504/802). Definite diagnostic yield 7.7% (62/802). Of diagnostic cases, 84% (52/62) had pathogenic/likely pathogenic **FAS** variants, equal to 6.5% of the full cohort (52/802). Diagnostic yield increased to 30% among patients also meeting abnormal ALPS immunology criteria. Among 37 unique diagnostic **FAS** variants, 46% (17/37) were novel. Domain distribution: 58% (30/52) of FAS-positive cases had heterozygous variants in the intracellular domain; of these, 57% (17/30) were missense variants in the death domain. | https://doi.org/10.1007/s10875-024-01772-z | Jul 2024 | (pqac-00000008, pqac-00000009) |
| GBM CAN008 / asunercept outcomes | Chang et al., *Biomedical Journal*; phase 1/2 open-label trial analysis in newly diagnosed glioblastoma | Trial NCT02853565 enrolled 10 patients; 9 evaluable. Dosing groups: 200 mg/week (n=3) and 400 mg/week (n=6) CAN008/asunercept plus standard CCRT. Compared with historical Taiwanese GBM cohort (n=164), high-dose CAN008 group showed OS at 2 and 5 years of 83% and 67% vs 40.1% and 8.8% in the historical cohort; historical cohort median OS 20 months; improved OS with high-dose CAN008, *p*=0.0103. Biomarker findings: low CD95L promoter CpG2 methylation associated with better response; better responders had higher variant count and tumor mutational burden. | https://doi.org/10.1016/j.bj.2023.100660 | Aug 2024 | (pqac-00000012) |
| MDS APG101 registry parameters | ClinicalTrials.gov NCT01736436; APG101/asunercept in transfusion-dependent low/intermediate-risk MDS | Phase 1, single-arm, open-label interventional study; enrollment 20. Intervention: APG101 100 mg IV weekly for 12 weeks with 6-month follow-up (37 weeks total). Primary objective/outcome: safety and tolerability (AEs/SAEs, ECGs, abdominal ultrasound, anti-drug antibodies, lymphocyte subsets/activation markers, ECOG). Secondary outcomes: overall survival, changes in transfusion frequency, bone marrow parameters (histologic/cytologic/cytogenetic), and hemoglobin levels. Key eligibility included adult WHO-classified de novo low/intermediate-risk MDS, blast count <5%, transfusion dependence, refractory/unlikely to respond to ESA. | https://clinicaltrials.gov/study/NCT01736436 | Registry 2013 | (pqac-00000020) |
| MDS APG101 molecular substudy | Streuer et al., *Annals of Hematology*; molecular follow-up from NCT01736436 | Bone marrow from 12 low-risk MDS patients analyzed at 58 time points by serial whole-exome sequencing. Mean 3.5 molecularly defined subclones per patient (range 2–6). Molecular response defined as dominant clone VAF decrease ≥10% occurred in 9/12 patients (75%); mean VAF decrease 20%, range 10.5–39.2%. Most clonal decline occurred after completion of 12 weeks treatment. Early pronounced reductions were noted in subclones with DNMT3A, IDH2, or TET2 mutations. | https://doi.org/10.1007/s00277-024-05664-5 | Feb 2024 | (pqac-00000013) |
| Severe COVID-19 trial design | ClinicalTrials.gov NCT04535674; asunercept in hospitalized severe COVID-19 | Multicenter, open-label, randomized, phase 2 trial; enrollment 438 (actual). Parallel arms: Standard of Care alone vs SoC + asunercept at 25 mg, 100 mg, or 400 mg weekly IV. Primary endpoint: time to sustained clinical improvement of at least 1 category on the 9-category WHO ordinal scale, sustained for 2 consecutive days, measured Days 1–29. Secondary endpoints included NEWS-based recovery/discharge, oxygenation-free days, ventilator-free days, hospital/ICU outcomes, and mortality at days 15, 29, 60, and 90. | https://clinicaltrials.gov/study/NCT04535674 | Registry 2020 | (pqac-00000018) |


*Table: This table compiles recent quantitative and clinical evidence for human FAS/CD95, spanning inherited disease genetics, oncology trials, hematology studies, and COVID-19 trial design. It is useful as a compact evidence map linking FAS biology to real-world human datasets and therapeutic development.*