| NC100403905 (Lachmann, 2009 #1833) | No RCT! [pivotal] | In a Phase III RCT |
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48-week three-part multicentre study, i.e. an 8-week open-label period (Part I), a 24-week randomised, DB, placebo-controlled withdrawal period (Part II)(각 n=15)(37y), followed by a 16-week OL period (Part III). Primary Outcome Measures □: 3. Percent of Participants With Disease Flare -[Disease Flare] i) CRP and/or SAA > 30 mg/L and ii) either a PGA > minimal, or PGA (equal to minimal and > minimal SD [PGA Physician's global assessment) ] urticarial rash, arthralgia, myalgia, headache/migraine, conjunctivitis, fatigue or malaise, and other symptoms related or unrelated to CAPS -The assessment was performed with the use of a 5-point scale for disease activity: absent, minimal, mild, moderate, or severe [Response] -Complete Response (CR): PGA and SD ≤ minimal and normal CRP and/or SAA -Partial Response (PR): a reduction of CRP and/or SAA from baseline (BL) by >30% but not reaching normal values and PGA improvement from BL by at least one category. -Nonresponders = no PR by Day 8 or no CR by Day 15. |
Study 03-AR-0298, Phase I/II, prospective, long-term, open-label and uncontrolled 43 adult and paediatric patients (36 patients aged 8 months to < 18 years) with severe CAPS (NOMID/CINCA and MWS),, Mean age: 10.3 (10.4)y Primary: 1. DSSS UPTO 3-6M (a disease-specific Diary Symptom Sum Score (DSSS)) DSSS included the sum of the 5 key symptoms fever, rash, joint pain, vomiting, and headache., 2. SAA (UPTO 3-6M) A rapid and sustained decrease in (SAA: 149→31 mg/L), (CRP: 51→ 9→ 6→ 4 mg/L) (ESR: 52→ 13 mm/h), and a normalization of inflammatory hematological changes are seen. In the severe form of CAPS, long-term treatment improves the systemic inflammatory organ manifestations of the eye, inner ear, and CNS. Hearing and visual acuity did not deteriorate further during anakinra treatment. | In a Phase III RCT comprising two sequential studies of 24 weeks in adults with CAPS (FCAS and MWS)15 followed by a 72-week open-label treatment extension, 16 rilonacept significantly reduced (84%) the symptom score versus placebo (13%; table 2),15 with reduction in the number of disease flare days, APRs and the limitations in patients' daily activities.15 16 |
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[HEARing] -There were small changes in hearing that stabilized at Month 12, indicating a subjective improvement in hearing. -More than 50% of the patients recorded no symptoms at baseline, and this subgroup showed no worsening with time, -the subgroup with symptoms recorded at baseline improved in hearing from a mean of 2.3 to 0.9 at Month 12. [vision] -there were only very minor changes and no indications of worsening in the subgroup with no vision loss at baseline (21 of 27 patients). -In the subgroup with vision loss present at baseline, there was a slight decrease in the self-perceived vision loss score from a mean value of 2.1 at baseline to 1.5 or lower at subsequent visits during the study. [ICP] (Normal: <200 mm,) 239 (15) bl → 212 (17) 3m → 183 (13) m36, → 179 (18) m60. 느리구나! 3년소요! [CSF WBC] 27.9 (5.7) cells/uL → 11.0 (2.8) m3, → 5.7 (1.0) m36, → 3.5 (0.6) m60 (normal 5이하) 느리구나! 3년소요! [MRI] xvi) leptomeningeal enhancement (active infla): present in 10 of 25 patients (40.0%) at baseline and in 1 of 19 patients (5.3%) at Month 60. → Eight patients with enhancement present at baseline improved at Month 36 and 7 patients at Month 60. 느리구나! 3년소요! One patient worsened at Month 36, but normalized and had no sign of enhancement at Month 60. xvii) ventricular enlargement: (chronic): 43.5% --> 41.2% (m60), Ventriculomegaly was present in 10 of 23 patients (43.5%) at baseline, and an additional 2 patients had shunts. As expected in these patients with long-standing disease, treatment had limited effect on this condition; a similar proportion of patients, 7 of 17 (41.2%), had ventriculomegaly at Month 60 as at baseline. (One patient with ventriculomegaly present at baseline improved at Month 36. This was a patient who started treatment at the age of 0.8 years (patient no 2042). No patient with normal ventricular appearance at baseline developed ventriculomegaly during treatment.) [ear] 구분: active) cochlear enhancement (만 빠르게 좋아심?) → hearing loss xviii) Of the 20 patients (87.0%) with some degree of cochlear enhancement on the initial MRI worst ear, 11 of 20 patients (57.9%) had normalized at Month 3. Four additional patients improved by at least 1 grade, totaling in 15 improved patients (78.9% of those with some degree of cochlear enhancement present at baseline). → At Month 36, 10 of 20 patients (50%) were free of cochlear enhancement in both ears and 9 of 19 patients (47.4%) at Month 60 (or last assessment after month 42). xix) From diary data recording subjective hearing, the subgroup with symptoms at baseline improved in self-perceived hearing from a mean score of 2.3 to 0.9 at Month 12. xx) By objective measures, this is reflected as small improvements in air conduction ePTA score for best ear compared to baseline early in treatment. xxi) Patients ≥12 years had more severe hearing - impairment than patients <12 years. The older patients in general stabilized without progressive worsening, but typically did not improve. Patients with normal hearing or limited ePTA abnormalities were younger at baseline and did not deteriorate with treatment over time. xxii) Hearing loss was not improved by anakinra treatment. [eye] 구분: active) uveitis/papilledema(도?)(만 빠르게 좋아짐?) → vision xxiii) full ophthalmologic examinations including visual acuity (presented as logMAR), visual field and dilated eye examinations. xxiv) Uveitis was present (assessed from "trace" to score "3") at baseline or had occurred historically in 8/17 patients. → During the study, none of these patients experienced any new events. One patient with no previous uveitis reported 2 events as AEs after 8 and 10 months of treatment; the inflammation resolved. Up to Month 60 there had been no other AE |
[results] Rash 거의 없앰. |
Uncertain Spans
| location | transcription | uncertainty |
|---|---|---|
| Lachmann cell trial id | NC100403905 | The expected NCT-prefix registry id (likely NCT00403905) is rendered as NC100403905 due to OCR-confused T vs 1; preserved as visible. |
| ear annotation | 만 빠르게 좋아심? | Final hangul character may be 짐 rather than 심; small font and Korean handwriting style. |
| Tx vision-outcome cell | Month 36 of -0.88 (95% CI -1.06 to -0.70; p-value <0.0001) in the best eye and ... | Trailing text is cut off at the bottom of this photo. |
| trial column 3 row label | column header for the rilonacept summary | The rilonacept block lacks a separate trial-id cell at the top; it appears merged with the second study’s row. |
| diary plot Y-axis | y-axis label of the 5-symptom diary line plot | The y-axis label is too small to read at this zoom. |