Combining treat-to-target principles with patient choice: A small step AHEAD in the right direction

IF 8.4 2区 医学 Q1 DERMATOLOGY Journal of the European Academy of Dermatology and Venereology Pub Date : 2024-10-25 DOI:10.1111/jdv.20298
Hywel C. Williams
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Abstract

The treat-to-target approach specifies optimum treatment targets such as blood glucose levels that result in health benefit. Setting single targets for complex inflammatory conditions such as atopic dermatitis (AD) is challenging, and depends on which disease aspects disease are considered to be important, by whom, along with how and when to measure them. A criticism of treat-to-target is its one-size-fits-all approach. Patients/carers may have different preferences on what is important for them, and what is a worthwhile gain when traded-off against the harms and inconvenience of particular treatments.

To address these limitations of a treat-to-target approach, the AHEAD (Aiming High in Eczema/Atopic Dermatitis) international group sought to identify what aspects of atopic dermatitis (AD) might be included in a treat-to-target approach and how they might be measured.1 The group's previous survey identified the most significant symptoms for adult AD patients, how they are measured and treatment expectations. The group used that data to draft a series of treatment recommendations that were subject to a Delphi survey involving 77 AD experts. Unusually, consensus was reached for all 34 recommendations after just one voting round. The group suggest that patients should identify between one to three from six features selected by the AD experts (itch, skin appearance, sleep disturbance, mental health, skin pain and daily life impact). The clinician also chooses an additional objective assessment of disease control. Targets for both optimal and moderate control are suggested for these outcomes for 3–6 months, and monitored with a recommended instrument chosen by the clinician.

What I liked about this initiative was the attempt to combine the best of both worlds—retaining more ambitious treatment targets for people with AD with some input from patients on what to measure and how. The proposed outcomes align with clinical practice outcomes from the Harmonising Outcomes Measures for Eczema (HOME) initiative.2 I also liked the flexibility for moderate as well as optimal control.

What was missing was lack of consideration of other crucial aspects that govern treatment choices for patients including adverse effects, inconvenience, access and costs. Although adverse effects may be treatment-specific, balancing benefits and harms is a mandatory part of shared decision-making as exemplified in AD patient decision aids.3 The preceding adult patient qualitative work, sponsored by AbbVie and cited only in abstract form, was useful but limited, as young people and children/carers where AD is commonest were not included. Although the adult qualitative study was considered when drafting the initial AHEAD recommendations, the complete absence of patient/carer involvement in the final recommendations was disappointing. Other initiatives such as HOME have found that working with patients/carers at consensus meetings fulfilling, even for complex issues such as disease measurement scales.

Lack of consensus often drives the need for consensus.4 The fact that consensus was achieved in just one round sounds fortuitous, but it came across like group think. There was little evidence of learning from dissenting voices, and the bar for defining agreement that included ‘mildly agree’ was quite low. The definition of treatment ‘success’ is also not clear given that patients can nominate up to three targets. Similarly, if a patient looks better according to >75% EASI reduction but deteriorates on their nominated symptom score, is that a ‘success’ or a case of looking better but feeling worse?5 Frequency of outcome assessment is not mentioned. The list of conflicts of interest of the group is impressive and runs into two pages. Whether such heavy ties with an industry that develops new systemic treatments have influenced the initiative is unclear.

AHEAD represent a step forward in combining ambitious treatment targets, patient choice and reliable outcomes. The next step is genuine rather than partial involvement of patients and carers of all ages and the creation of decision aids that that trade-off ambitious treatment targets with potential harms.

The author is a member of the executive of the Harmonising Outcome Measures for Eczema (HOME) initiative that has proposed core outcomes for atopic dermatitis research and clinical practice cited in this article. He has no conflicts with the pharmaceutical industry.

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将靶向治疗原则与患者选择相结合:朝着正确方向迈出的一小步。
针对目标治疗的方法规定了最佳治疗目标,如能带来健康益处的血糖水平。为特应性皮炎(AD)等复杂炎症设定单一目标具有挑战性,这取决于疾病的哪些方面被认为是重要的、由谁认为是重要的,以及如何和何时对其进行测量。对 "靶向治疗 "的批评在于其 "一刀切 "的方法。患者/照护者可能对哪些因素对他们来说是重要的有不同的偏好,在与特定治疗的危害和不便进行权衡时,哪些因素是值得获得的。为了解决 "对症下药 "方法的这些局限性,AHEAD(Aiming High in Eczema/Atopic Dermatitis)国际小组试图确定特应性皮炎(AD)的哪些方面可以纳入 "对症下药 "方法,以及如何对其进行衡量。该小组之前的调查确定了成人特应性皮炎患者最主要的症状、如何衡量这些症状以及治疗期望。该小组利用这些数据起草了一系列治疗建议,并对 77 位注意力缺失症专家进行了德尔菲调查。不同寻常的是,仅经过一轮投票,所有 34 项建议都达成了共识。专家组建议,患者应从注意力缺失症专家选出的六项特征(瘙痒、皮肤外观、睡眠障碍、心理健康、皮肤疼痛和对日常生活的影响)中找出一至三项。临床医生还可选择对疾病控制情况进行额外的客观评估。我喜欢这项倡议的原因是它试图将两方面的优点结合起来--既为AD患者保留更宏伟的治疗目标,又让患者就测量什么和如何测量提出一些意见。建议的结果与 "湿疹统一结果测量(HOME)倡议 "2 的临床实践结果相一致。我还喜欢该倡议在适度控制和最佳控制方面的灵活性。尽管不良反应可能与治疗有关,但平衡益处和危害是共同决策中必须考虑的部分,AD 患者决策辅助工具就是一个例子。3 之前由艾伯维赞助、仅以摘要形式引用的成人患者定性研究非常有用,但也很有限,因为其中并未包括 AD 最常见的年轻人和儿童/护理人员。虽然在起草最初的 AHEAD 建议时考虑了成人定性研究,但最终建议中完全没有患者/护理者的参与,这一点令人失望。其他倡议,如 "居家"(HOME),发现在共识会议上与患者/护理者的合作,即使是在疾病测量量表等复杂的问题上,也能取得成效。几乎没有证据表明从不同意见中汲取了什么教训,而且对包括 "略表同意 "在内的一致意见的定义标准也相当低。治疗 "成功 "的定义也不明确,因为患者最多可以提名三个目标。同样,如果患者的 EASI 降低了 75%,看起来有所好转,但其指定的症状评分却恶化了,这究竟是 "成功 "还是看起来好转但感觉更糟?该小组的利益冲突清单长达两页,令人印象深刻。AHEAD代表了将雄心勃勃的治疗目标、患者的选择和可靠的结果结合起来的一个进步。下一步是让所有年龄段的患者和照护者真正而非部分地参与进来,并创建决策辅助工具,以权衡雄心勃勃的治疗目标和潜在的危害。作者是 "湿疹结果衡量标准协调计划"(HOME)的执行成员之一,该计划为本文引用的特应性皮炎研究和临床实践提出了核心结果。他与制药业没有冲突。
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来源期刊
CiteScore
10.70
自引率
8.70%
发文量
874
审稿时长
3-6 weeks
期刊介绍: The Journal of the European Academy of Dermatology and Venereology (JEADV) is a publication that focuses on dermatology and venereology. It covers various topics within these fields, including both clinical and basic science subjects. The journal publishes articles in different formats, such as editorials, review articles, practice articles, original papers, short reports, letters to the editor, features, and announcements from the European Academy of Dermatology and Venereology (EADV). The journal covers a wide range of keywords, including allergy, cancer, clinical medicine, cytokines, dermatology, drug reactions, hair disease, laser therapy, nail disease, oncology, skin cancer, skin disease, therapeutics, tumors, virus infections, and venereology. The JEADV is indexed and abstracted by various databases and resources, including Abstracts on Hygiene & Communicable Diseases, Academic Search, AgBiotech News & Information, Botanical Pesticides, CAB Abstracts®, Embase, Global Health, InfoTrac, Ingenta Select, MEDLINE/PubMed, Science Citation Index Expanded, and others.
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