Assessing the validity, responsiveness and meaningfulness of the Hidradenitis Suppurativa Clinical Response (HiSCR) as the clinical endpoint for hidradenitis suppurativa treatment

Kimball, Alexa B.; Jemec, Gregor B. E.; Yang, Min; Kageleiry, Andrew; Signorovitch, James; Okun, Martin; Gu, Yihua; Wang, K.; Mulani, Parvez; Sundaram, Murali

doi:10.1111/bjd.13270

Cited by 171 publications

(183 citation statements)

References 18 publications

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“…The primary outcome was clinical response, measured in the PIONEER trials using the HiSCR measure developed by the company. Clinical advice received by the ERG confirmed that HiSCR had been validated but, in terms of clinical decision-making, its findings should be viewed alongside patientreported outcome measures (PROMs), in particular the DLQI and a pain measure [17,21]. The ERG agreed with the company that the M10-467 trial was at low risk of bias for the relevant Period 1 (up to week 16).…”

Section: Critique Of Clinical Effectiveness Evidence and Interpretationmentioning

confidence: 87%

“…The clinical evidence consisted of three separate reviews: (1) a review of clinical efficacy evidence from RCTs of treatments for HS, specifically trials comparing adalimumab with placebo in adults with moderate-to-severe HS: a Phase II "dosing" trial, M10-467 [17], and two Phase III trials, PIONEER I and II [18,19]; (2) a review of evidence from a non-controlled open-label extension (OLE) study (M12-555) [20], and; (3) a review of safety evidence from the RCTs and the OLE study.…”

Section: Clinical Evidence Submitted By the Companymentioning

confidence: 99%

“…Significant improvements were also observed at week 16 in individual symptoms, overall disease severity and pain scores for adalimumab 40mg EW compared with placebo. In the PIONEER trials, clinical response was evaluated by Hidradenitis Suppurativa Clinical Response (HiSCR), defined as ≥50% reduction in the total abscess and inflammatory nodule [AN] count with no increase in abscess count or draining fistula count [17].…”

Section: Clinical Evidence Submitted By the Companymentioning

confidence: 99%

“…The ICER for adalimumab was greater than £30,000 per QALY gained in four scenario analyses: (i) time horizon=20 years; (ii) use of PIONEER II data only; (iii) use of last state carried forward imputation, and; (iv) discontinuation rate for adalimumab non-responders after week 36 based on the OLE study. (ii) The HiSCR validation study reported by Kimball et al relates specifically to the full HiSCR threshold (≥50% reduction in ANs, with no increase in abscesses or draining fistulas from baseline) [17]. Kimball et al reported that patients with worsening disease or minimal improvement in ANs (<30% reduction) did not have a meaningful improvement on the DLQI and reported some worsening in pain despite improvements in total work impairment and total activity impairment.…”

Section: Cost-effectiveness Evidence Submitted By the Companymentioning

confidence: 99%

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Adalimumab for Treating Moderate-to-Severe Hidradenitis Suppurativa: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

et al. 2017

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As part of its Single Technology Appraisal (STA) process, the National Institute for Health and Care Excellence (NICE) invited the manufacturer of adalimumab (AbbVie) to submit evidence on the clinical effectiveness and cost-effectiveness of adalimumab for the treatment of moderate-to-severe hidradenitis suppurativa (HS). The appraisal assessed adalimumab as monotherapy in adult patients with an inadequate response to conventional systemic HS therapy. The School of Health and Related Research Technology Appraisal Group was commissioned to act as the independent Evidence Review Group (ERG). The ERG produced a critical review of the evidence for the clinical effectiveness and cost-effectiveness of the technology, based on the company's submission to NICE. The evidence was mainly derived from three randomised controlled trials comparing adalimumab with placebo in adults with moderate-to-severe HS. The clinical effectiveness review found that significantly more patients achieved a clinical response in the adalimumab groups than the control groups, but that the treatment effect varied between trials and there was uncertainty regarding its impact on a range of other relevant outcomes, as well as long-term efficacy. The company's submitted Markov model assessed the incremental cost-effectiveness of adalimumab versus standard care for the treatment of HS from the perspective of the National Health Service (NHS) and Personal Social Services (PSS) over a lifetime horizon. The original submitted model, which included a Patient Access Scheme (PAS), suggested that the incremental cost-effectiveness ratio (ICER) for adalimumab versus standard care is expected to be £16,162 per QALY gained. Following a critique of the model, the ERG's preferred base case, which corrected programming errors and structural problems surrounding discontinuation rules, and incorporated a lower unit cost for HS surgery, resulted in a probabilistic ICER of £29,725 per QALY gained. Based on additional analyses undertaken by the company and the ERG following the publication of the Appraisal Consultation Document (ACD), the appraisal committee concluded that the maximum possible ICER for adalimumab compared with supportive care was between £28,500 and £33,200 per QALY gained, but was likely to be lower. The appraisal committee recommended adalimumab (with the PAS) for the treatment of active moderate-to-severe HS in adults whose disease has not responded to conventional systemic therapy.

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Section: Critique Of Clinical Effectiveness Evidence and Interpretationmentioning

confidence: 87%

Section: Clinical Evidence Submitted By the Companymentioning

confidence: 99%

Section: Clinical Evidence Submitted By the Companymentioning

confidence: 99%

Section: Cost-effectiveness Evidence Submitted By the Companymentioning

confidence: 99%

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Adalimumab for Treating Moderate-to-Severe Hidradenitis Suppurativa: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

et al. 2017

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“…2). Существует также оценка степени тяжести заболевания по индексу PGA (Physician's Global Assessment -глобальная оценка вра-ча) [29] (рис. 1).…”

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Hidradenitis Suppurativa: Modern Concepts of Pathogenesis and Therapy; Successful Experience of Adalimumab Therapy

Хобейш

Шустов

Sokolovsky

et al. 2017

Vestnik dermatologii i venerologii

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Гидраденит суппуративный:современные представления о патогенезе, терапии и успешный опыт лечения адалимумабом Хобейш М. М., Шустов Д. В., Соколовский Е. В.Первый Санкт-Петербургский государственный медицинский университет им. акад. И. П. Павлова Минздрава России 197022, Российская Федерация, г. Санкт-Петербург, ул. Льва Толстого, д. 6/8, корп. 4Гидраденит cуппуративный (ГС) -хроническое воспалительное кожное заболевание, поражающее преимущественно участки расположения апокриновых желез; характеризующееся рецидивирующими болезненными узлами, абсцессами, приводящими к образованию свищей и рубцеванию. Современные представления о патогенезе ГС позволяют рассматривать его как разновидность инверсных акне, так как механизм развития ГС и инверсных акне не различается. Детальные исследования пациентов с ГС, проведенные в последние годы, дали возможность ряду авторов предположить, что ГC является системным хроническим иммуноопосредованным прогрессирующим заболеванием. Определена ключевая роль провоспалительного цитокина фактора некроза опухоли (ФНО-) в развитии воспалительного процесса при ГC. Повышенные уровни ФНО-обнаружены в крови и в очагах поражения ГС, причем определяется положительная корреляция уровня ФНО-с тяжестью болезни. В настоящее время в качестве терапии 1-й линии при отсутствии адекватного ответа на стандартную системную терапию в случае активного ГС средней или тяжелой степени тяжести рекомендовано лечение блокатором ФНО--адалимумабом, единственным на сегодняшний день генно-инженерным биологическим препаратом, одобренным для лечения ГС/инверсных акне в мире и в России. Представленный клинический случай подтверждает тот факт, что для пациентов с ГС очень важно максимально быстрое определение правильного диагноза и назначение рационального лечения в соответствии с разработанными рекомендациями. Ошибочные диагнозы, неадекватная терапия (хирургическое лечение, короткие курсы приема антибиотиков и т.д.) способствуют прогрессированию заболевания, появлению общих симптомов за счет присоединения вторичной инфекции и повышению общей интоксикации, ограничению работоспособности, возникновению стойких и грубых рубцов, деформаций пораженных участков кожи. Наш клинический случай применения адалимумаба ( умиры ) у пациента с крайне тяжелой формой ГС показал, что лечение этим препаратом может быть эффективным и хорошо переносимым. Hidradenitis suppurativa (HS) is a chronic inflammatory skin disease that mainly affects the sites of the apocrine glands; HS is characterised by relapsing painful knots and abscesses, leading to the formation of fistulas and scarring. Modern ideas about the pathogenesis of HS allow us to consider it as a kind of inverse acne, since the mechanism of development of HS and inverse acne does not differ. Detailed studies of patients with HS in recent years have provided the basis for several authors to suggest that HS is a systemic, chronic, immune-mediated progressive disease. The key role of the proinflammatory cytokine tumor necrosis factor (TNF-) in the development of the inflammatory process ...

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Safety and Efficacy of Anakinra in Severe Hidradenitis Suppurativa

et al. 2016

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IMPORTANCE Hidradenitis suppurativa (HS) is a common skin disorder in which excessive inflammation is believed to have an important role. There is no specific therapy for HS. OBJECTIVE To investigate the safety and efficacy of the anti-inflammatory biological therapy anakinra in HS. DESIGN, SETTING, AND PARTICIPANTS Double-blind, randomized, placebo-controlled clinical trial with a 12-week treatment phase and a 12-week follow-up phase. The setting was Attikon University General Hospital, a tertiary care institution in Athens, Greece. Participants were 20 patients with Hurley stage II or III HS. The study and the analysis were conducted between

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Assessing the validity, responsiveness and meaningfulness of the Hidradenitis Suppurativa Clinical Response (HiSCR) as the clinical endpoint for hidradenitis suppurativa treatment

Cited by 171 publications

References 18 publications

Adalimumab for Treating Moderate-to-Severe Hidradenitis Suppurativa: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

Adalimumab for Treating Moderate-to-Severe Hidradenitis Suppurativa: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

Hidradenitis Suppurativa: Modern Concepts of Pathogenesis and Therapy; Successful Experience of Adalimumab Therapy

Safety and Efficacy of Anakinra in Severe Hidradenitis Suppurativa

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