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Peak nasal inspiratory flow assessment of polyp size and response from SYNAPSE 鼻腔吸气流量峰值评估息肉大小和 SYNAPSE 反应
Pub Date : 2024-08-20 DOI: 10.1016/j.jacig.2024.100327
Amber U. Luong MD, PhD , Joshua M. Levy MD, MPH, MSc , Ludger Klimek MD, PhD , Richard J. Harvey MD , Jared Silver MD, PhD , Steven G. Smith PhD , Abby Fuller MSc , Robert Chan MD , Peter W. Hellings MD, PhD

Background

In the phase III SYNAPSE study, mepolizumab plus standard of care reduced total endoscopic nasal polyp score (NPS) versus that with placebo in patients with chronic rhinosinusitis with nasal polyps.

Objective

Our aim was to investigate relationships between NPS and (1) peak nasal inspiratory flow (PNIF) and (2) patient-reported outcomes.

Methods

In this post hoc analysis, patients randomized 1:1 received mepolizumab, 100 mg, or placebo subcutaneously every 4 weeks (plus standard of care). Changes from baseline in PNIF (week 52), visual analog scale scores (overall symptoms, nasal obstruction, and loss of smell [weeks 49-52]), and total 22-Item Sino-Nasal Outcome Test score (week 52) were assessed in patients with or without improvements in NPS (improvement of ≥1 point) or without (improvement of <1 point or worsening).

Results

Patients with improvements in NPS had greater improvements in PNIF (a median of 50 L per minute [interquartile range (IQR) = 10.5-87.5] with mepolizumab vs a median of 40 L per minute [IQR = 0-85.0] with placebo) than did those patients without improvements in NPS (a median of 0.0 L per minute [IQR = –10.0 to 45.0] with mepolizumab vs a median of 0.0 L per minute [IQR = –30.0 to 30.0] with placebo). Similar results were seen for the following: change from baseline in overall symptoms (a median of –5.8 [IQR = –8.1 to –3.80] with mepolizumab and a median of –4.1 [IQR = –7.0 to –1.2] with placebo with improvement in NPS vs a median of –1.3 [IQR = –6.3 to 0.0] with mepolizumab and a median of –0.1 [IQR = –3.4 to 0.0] with placebo without improvement in NPS); change in nasal obstruction (a median of –5.7 [IQR = –8.2 to –3.5] with mepolizumab and a median of –4.5 [IQR = –7.3 to –1.2] with placebo with improvement in NPS vs a median of –1.3 [IQR = –6.6 to 0.0] with mepolizumab and a median of 0.0 [IQR = –3.6 to 0.0] with placebo without improvement in NPS); change in loss of smell (a median of –2.8 [IQR = –7.9 to 0.0] with mepolizumab and a median of –0.7 [IQR = –4.0 to 0.0] with placebo with improvement in NPS vs a median of 0.0 [IQR = –2.4 to 0.0] with mepolizumab and a median of 0.0 [IQR = –0.3 to 0.0]) with placebo without improvement in NPS); and change in visual analog scale score and 22-Item Sino-Nasal Outcome Test total score (a median of –37.0 [IQR = –52.0 to –24.0] with mepolizumab and a median of –29.0 [IQR = –43.0 to –9.0] with placebo with improvement in NPS vs a median of –16.0 [IQR = –42.0 to 0.0] with mepolizumab and a median of 0.0 [IQR = –27.0 to 0.0] with placebo without improvement in NPS).

Conclusion

Improvement in NPS was associated with improvements in PNIF and patient-reported outcomes irrespective of treatment. PNIF could be a useful noninvasive tool for monitoring nasal polyp size.
背景在III期SYNAPSE研究中,对于伴有鼻息肉的慢性鼻窦炎患者,美泊利珠单抗加标准治疗与安慰剂相比可降低内窥镜鼻息肉总评分(NPS)。我们的目的是研究 NPS 与 (1) 鼻吸气流量峰值 (PNIF) 和 (2) 患者报告结果之间的关系。方法在这项事后分析中,患者按 1:1 随机分配,每 4 周一次皮下注射 100 毫克的麦泊利单抗或安慰剂(加上标准护理)。在NPS改善或未改善(改善≥1分)或未改善(改善<1分或恶化)的患者中,评估PNIF(第52周)、视觉模拟量表评分(总体症状、鼻阻塞和嗅觉丧失[第49-52周])和22项中鼻结果测试总分(第52周)与基线相比的变化。结果与安慰剂相比,NPS改善的患者的PNIF改善幅度更大(使用mepolizumab的中位数为每分钟50升[四分位距(IQR)=10.5-87.5],而使用安慰剂的中位数为每分钟40升[IQR=0-85.中位数为每分钟 0.0 升[IQR = -10.0 至 45.0],而安慰剂的中位数为每分钟 0.0 升[IQR = -30.0 至 30.0])。以下方面也出现了类似的结果:总体症状与基线相比的变化(使用 mepolizumab 的中位数为 -5.8 [IQR = -8.1 至 -3.80],使用安慰剂的中位数为 -4.1 [IQR = -7.0 至 -1.2] ,NPS 有所改善;使用 mepolizumab 的中位数为 -1.3 [IQR = -6.3 至 0.0],使用安慰剂的中位数为 -0.1 [IQR = -3.4 至 0.0],NPS 没有改善。0],而安慰剂的中位数为-0.1[IQR = -3.4至0.0],NPS没有改善);鼻阻塞的变化(使用mepolizumab的中位数为-5.7[IQR = -8.2至-3.5],而使用安慰剂的中位数为-4.5[IQR = -7.3至-1.2],而安慰剂的中位数为-1.3[IQR = -6.6 to 0.0],安慰剂的中位数为0.0[IQR = -3.6 to 0.0],NPS没有改善);嗅觉丧失的变化(mepolizumab的中位数为-2.8[IQR = -7.9至0.0],安慰剂的中位数为-0.7[IQR = -4.0至0.0],NPS有所改善;与之相比,甲泼尼单抗的中位数为0.0[IQR = -2.4至0.0],安慰剂的中位数为0.0[IQR=-0.3至0.0]),安慰剂则无改善);以及视觉模拟量表评分和22项鼻结果测试总分的变化(中位数为-37.0[IQR=-52.0至-24.结论无论治疗方法如何,NPS 的改善与 PNIF 和患者报告结果的改善相关。PNIF可能是监测鼻息肉大小的一种有用的无创工具。
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引用次数: 0
Agaricus bisporus mushroom anaphylaxis: A case report and review of the literature 双孢蘑菇过敏性休克:病例报告和文献综述
Pub Date : 2024-08-12 DOI: 10.1016/j.jacig.2024.100324
Syed B. Ali MBBS, FRACP , William Smith MBBS, FRACP, PhD

Background

Anaphylaxis in response to edible mushrooms is uncommon, and sensitization may be independent of occupational exposure. Globally, Agaricus bisporus is one the most cultivated mushroom species, comprising the button, portobello, and Swiss brown varietals.

Objective

Our aim was to describe a case of anaphylaxis in an older nonatopic patient following ingestion of A bisporus and undertake a review of the literature.

Methods

A 77-year-old female was referred to the allergy clinic with a history of grade 3 hypotensive anaphylaxis following ingestion of a mushroom omelette. Her tryptase level 4 hours following acute symptoms was elevated. She continued to consume all other omelette ingredients with the exception of mushroom. Additional investigations included specific IgE testing as well as comprehensive skin prick testing (SPT) to raw and boiled mushrooms.

Results

One month after her index reaction, the result of SPT to boiled button mushroom was positive. The patient’s baseline tryptase level was normal. The results of further SPT with other commonly available boiled mushrooms (the A bisporus mushrooms portobello and Swiss brown, as well as enoki, oyster, and shiitake mushrooms) were also positive.

A review of the literature revealed 27 other cases, with an average patient age of 28.8 years (range 8-68 years) and no gender predisposition. Cases from Europe (n = 14, 51.8%) and Asia (n = 12, 44.4%) comprised the majority of the reports. A bisporus was reported in more than one-third of the cases (n = 10, 37.1%) from Australia, India, Portugal, Spain, and the United Kingdom. Cross-sensitization among the edible mushroom species was evident for those individuals who were tested, but no clear and consistent pattern was identified. In some cases, further diagnostics identified α-mannosidase, α-trehalose, mannitol, and ribosomal protein as specific mushroom allergens.

Conclusion

Clinicians should be aware of mushroom as a cause for anaphylaxis, especially in nonatopic and de novo presentations.

背景食用菌引起的过敏性休克并不常见,过敏可能与职业接触无关。在全球范围内,双孢蘑菇是栽培最多的蘑菇品种之一,包括金针菇、波多贝罗蘑菇和瑞士褐蘑菇。我们的目的是描述一例老年非特异性患者在摄入双孢蘑菇后发生过敏性休克的病例,并对文献进行回顾。急性症状出现 4 小时后,她的胰蛋白酶水平升高。她继续食用除蘑菇外的所有其他煎蛋配料。其他检查包括特异性 IgE 检测以及对生蘑菇和水煮蘑菇的全面皮肤点刺试验(SPT)。患者的基线色氨酸酶水平正常。对其他常见水煮蘑菇(双孢蘑菇、波多贝罗蘑菇、瑞士褐蘑菇、金针菇、杏鲍菇和香菇)的进一步 SPT 结果也呈阳性。来自欧洲(14 例,占 51.8%)和亚洲(12 例,占 44.4%)的病例占报告病例的大多数。在来自澳大利亚、印度、葡萄牙、西班牙和英国的病例中,超过三分之一的病例(10 例,37.1%)患有双孢菇。在接受检测的个体中,食用菌种类之间的交叉致敏现象很明显,但没有发现明确一致的模式。在某些病例中,进一步诊断发现α-甘露糖苷酶、α-曲哈糖、甘露醇和核糖体蛋白是特异性蘑菇过敏原。
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引用次数: 0
The common cold: The need for an effective treatment amid the FDA discussion on oral phenylephrine 普通感冒:在美国食品及药物管理局讨论口服苯肾上腺素之际,需要一种有效的治疗方法
Pub Date : 2024-08-07 DOI: 10.1016/j.jacig.2024.100318
Emina Išerić BSc , Joris C. Verster PhD

An episode of the common cold can have a significant negative impact on quality of life, mood, and daily activities. In line with this fact, there is a growing demand for health care and treatments associated with the common cold. Current treatments aim to (1) inhibit symptom severity and (2) shorten the duration of an episode of the common cold. These products include analgesics, antihistamines, and decongestants. In addition, various supplements, including vitamins, minerals, and herbs, are marketed to treat the common cold. The current products marketed for treating the common cold may reduce the severity of some (but not all) common cold symptoms, but they usually do not shorten the common cold episode. The recent indication that phenylephrine is not effective means that it will ultimately need to be removed from the over-the-counter monograph. Manufacturers will consequently need to reformulate their products and withdraw oral phenylephrine-containing products. Several newly developed common cold products are currently under investigation. These clinical trials should evaluate their efficacy and safety, as there remains a clear need for common cold products that significantly reduce both the symptom severity and the duration of episodes of the common cold.

普通感冒的发作会对生活质量、情绪和日常活动产生严重的负面影响。因此,人们对普通感冒的保健和治疗需求日益增长。目前的治疗方法旨在:(1)抑制症状的严重程度;(2)缩短普通感冒发作的持续时间。这些产品包括镇痛剂、抗组胺剂和减充血剂。此外,市场上还有各种治疗普通感冒的补充剂,包括维生素、矿物质和草药。目前市场上销售的治疗普通感冒的产品可能会减轻一些(但不是全部)普通感冒症状的严重程度,但通常不会缩短普通感冒的发作时间。最近有迹象表明苯肾上腺素无效,这意味着最终需要将其从非处方药专论中删除。因此,生产商需要重新配制产品,并撤下含有苯肾上腺素的口服产品。目前正在对几种新开发的普通感冒产品进行调查。这些临床试验应评估其疗效和安全性,因为目前仍明显需要能显著降低普通感冒症状严重程度和缩短发病时间的普通感冒产品。
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引用次数: 0
Daratumumab-based treatment of monoclonal gammopathy–associated angioedema due to acquired C1-inhibitor deficiency 基于达拉单抗治疗获得性 C1 抑制剂缺乏引起的单克隆抗体病相关性血管性水肿
Pub Date : 2024-08-05 DOI: 10.1016/j.jacig.2024.100322
Remy S. Petersen MD , Lauré M. Fijen MD, PhD , Laurens E. Franssen MD, PhD , Josephine M.I. Vos MD, PhD , Danny M. Cohn MD, PhD

Daratumumab-based treatment could control severe, treatment-refractory, life-threatening angioedema due to acquired C1-inhibitor deficiency associated with monoclonal gammopathy.

基于达拉土单抗的治疗可以控制严重的、治疗难治的、危及生命的血管性水肿,这种水肿是由于后天C1抑制剂缺乏并伴有单克隆抗体病引起的。
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引用次数: 0
Running into trouble with soy: A case report and review of our shopping carts 大豆带来的麻烦病例报告和购物车回顾
Pub Date : 2024-08-05 DOI: 10.1016/j.jacig.2024.100321
Fionnuala Cox BA Hons, MB, BCh, BAO, MRCPI, FRCPath, PhD, Khairin Khalib MBBCh, MRCPI, FRCPath, Mary Keogan MD, FRCPI, FRCPath

Soy-dependent exercise-induced anaphylaxis is likely underdiagnosed and potentially on the rise. As soy gains popularity in Western diets, we highlight it as a hidden allergen in a variety of processed foods, including those marketed toward exercise enthusiasts.

大豆依赖性运动诱发过敏性休克很可能诊断不足,而且有可能呈上升趋势。随着大豆在西方饮食中的普及,我们强调它是各种加工食品(包括面向运动爱好者销售的食品)中隐藏的过敏原。
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引用次数: 0
Successful treatment of the dupilumab-induced psoriatic dermatitis/arthritis and atopic dermatitis with a JAK inhibitor: A case report and literature review 用 JAK 抑制剂成功治疗杜必鲁单抗诱发的银屑病皮炎/关节炎和特应性皮炎:病例报告和文献综述
Pub Date : 2024-08-05 DOI: 10.1016/j.jacig.2024.100323
Misuzu Tsunoda MD , Takeya Adachi MD, PhD , Yuuri Nakajima MD , Yoshihiro Yatomi MD , Tomoko Shimizu MD, PhD , Katsuki Nakasute MD

Dupilumab-induced psoriatic dermatitis and arthritis in a patient with atopic dermatitis were effectively managed with upadacitinib, highlighting the use of Janus kinase inhibitors as a possible treatment for biologic therapy side effects.

一位特应性皮炎患者因使用杜匹单抗引发的银屑病皮炎和关节炎,在使用达帕替尼后得到了有效控制,这凸显了使用Janus激酶抑制剂治疗生物疗法副作用的可能性。
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引用次数: 0
Rash decisions: Unmasking a risk phenotype in adults with persistent delayed penicillin allergy sensitized during historic infection with Epstein-Barr virus 皮疹决策:揭示历史上曾感染 Epstein-Barr 病毒并对其过敏的持续性迟发性青霉素过敏成人的风险表型
Pub Date : 2024-08-05 DOI: 10.1016/j.jacig.2024.100320
Fionnuala Cox BA Hons, PhD, MB, BCh, BAO, MRCPI, FRCPath , Natasha E. Holmes MBBS, PhD , Jamie Lee Waldron MD , Jason A. Trubiano MBBS, PhD

Background

Penicillin-associated exanthems in the setting of infectious mononucleosis caused by Epstein-Barr virus (EBV) are often viewed as a transient event, not a true allergy. Recent evidence challenges this and suggests that a notable subset of patients retain penicillin hypersensitivity.

Objective

We investigated the occurrence and predictors of persistent adulthood hypersensitivity in those with penicillin-associated rash occurring in the setting of EBV infection.

Methods

Retrospective analysis of data of patients referred for penicillin allergy testing to an Australian tertiary-care hospital captured from 2015 to 2023 was carried out.

Results

Of 2066 patients, 23 (1%) had penicillin-associated rash during an historic EBV infection; 16 (70%) were female; and median (interquartile range) age was 18 (16-20) years at index reaction and 38 (33.5-57) years at allergy testing. Skin prick testing and delayed intradermal testing to a penicillin panel were performed, followed by oral provocation challenge in those testing negative. Persistent sensitization was shown in 6 (26%) of 23; 4 (67%) of 6 positive delayed intradermal testing; and 3 (50%) of 6 had positive oral challenge test. Notably, 5 (83%) of 6 had a severe maculopapular exanthem with facial swelling, including 2 (33%) of 6 with probable drug reaction with eosinophilia and systemic symptoms (aka DRESS) during the index reaction, compared to 0 of 17 in patients tolerating penicillin on reexposure.

Conclusion

This study highlights the requirement of allergy testing in adult patients reporting a penicillin-associated severe maculopapular exanthem in the setting of EBV, even if it occurred during childhood or adolescence.

背景由 Epstein-Barr 病毒(EBV)引起的传染性单核细胞增多症中出现的青霉素相关外显子通常被视为一种短暂的现象,而非真正的过敏。我们研究了在 EBV 感染时出现青霉素相关皮疹的患者成年后持续过敏的发生率和预测因素。结果 在 2066 名患者中,23 人(1%)在历史性 EBV 感染期间出现青霉素相关皮疹;16 人(70%)为女性;指数反应时的中位年龄(四分位数间距)为 18(16-20)岁,过敏试验时的中位年龄为 38(33.5-57)岁。对患者进行了皮肤点刺试验和青霉素延迟皮内试验,对试验阴性的患者进行了口服激发试验。23 人中有 6 人(26%)出现持续过敏;6 人中有 4 人(67%)延迟皮内试验呈阳性;6 人中有 3 人(50%)口服激发试验呈阳性。值得注意的是,6 人中有 5 人(83%)出现严重的斑丘疹并伴有面部肿胀,包括 6 人中有 2 人(33%)在指数反应期间出现伴有嗜酸性粒细胞增多和全身症状的可能药物反应(又称 DRESS),而在再次接触青霉素的 17 名患者中,只有 0 人能够耐受。结论:本研究强调,对于报告与青霉素相关的严重斑丘疹性外伤的 EBV 成人患者,即使发生在儿童或青少年时期,也必须进行过敏检测。
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引用次数: 0
Aims and scope 目标和范围
Pub Date : 2024-08-01 DOI: 10.1016/S2772-8293(24)00103-6
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引用次数: 0
Biomarkers and patient-related factors associated with clinical outcomes in dupilumab-treated atopic dermatitis 与杜匹单抗治疗特应性皮炎临床疗效相关的生物标记物和患者相关因素
Pub Date : 2024-07-31 DOI: 10.1016/j.jacig.2024.100317
Makiko Kido-Nakahara MD , Daisuke Onozuka PhD , Kenji Izuhara PhD , Hidehisa Saeki MD , Satoshi Nunomura PhD , Motoi Takenaka MD , Mai Matsumoto MD , Yoko Kataoka MD , Rai Fujimoto MD , Sakae Kaneko MD , Eishin Morita MD , Akio Tanaka MD , Michihiro Hide MD , Tatsuro Okano MD , Tomomitsu Miyagaki MD , Natsuko Aoki MD , Kimiko Nakajima MD , Susumu Ichiyama MD , Kyoko Tonomura MD , Yukinobu Nakagawa MD , Takeshi Nakahara MD

Background

Atopic dermatitis (AD) is a common chronic eczematous skin disease with severe pruritus. Several new therapeutic agents for AD such as dupilumab, an anti–IL-4Rα antibody, have been developed in recent years. We need to predict which agent is the best choice for each patient, but this remains difficult.

Objective

Our aim was to examine clinical background factors and baseline biomarkers that could predict the achievement of improved clinical outcomes in patients with AD treated with dupilumab.

Methods

A multicenter, prospective observational study was conducted on 110 patients with AD. The Eczema Area and Severity Index was used as an objective assessment, and the Patient-Oriented Eczema Measure and Numerical Rating Scale for Pruritus were used as patient-reported outcomes. In addition, some clinical background factors were evaluated.

Results

The achievement of an absolute Eczema Area and Severity Index of 7 or less was negatively associated with current comorbidity of food allergy and baseline serum lactate dehydrogenase (LDH) levels. There were negative associations between achievement of a Patient-Oriented Eczema Measure score of 7 or less and duration of severe AD and between achievement of an itching Numerical Rating Scale for Pruritus score of 1 or less and current comorbidity of allergic conjunctivitis or baseline serum periostin level. Furthermore, signal detection analysis showed that a baseline serum LDH level less than 328 U/L could potentially be used as a cutoff value for predicting the efficacy of dupilumab.

Conclusion

Baseline biomarkers such as LDH and periostin and clinical background factors such as current comorbidity of food allergy and a long period of severe disease may be useful indicators when choosing dupilumab for systemic treatment for AD, as they can predict the efficacy of dupilumab.

背景特应性皮炎(AD)是一种常见的慢性湿疹性皮肤病,伴有严重的瘙痒。近年来开发出了几种治疗 AD 的新药,如抗 IL-4Rα 抗体 dupilumab。我们需要预测哪种药物是每位患者的最佳选择,但这仍然很困难。我们的目的是研究临床背景因素和基线生物标志物,以预测接受杜比鲁单抗治疗的AD患者临床疗效的改善情况。研究采用湿疹面积和严重程度指数作为客观评估指标,以患者为导向的湿疹测量法和瘙痒数字评定量表作为患者报告的结果。结果湿疹面积和严重程度指数绝对值达到或低于 7 与当前食物过敏合并症和基线血清乳酸脱氢酶 (LDH) 水平呈负相关。以患者为导向的湿疹测量(Patient-Oriented Eczema Measure)评分达到或低于 7 分与严重 AD 的持续时间呈负相关,瘙痒数字评分量表(Numerical Rating Scale for Pruritus)评分达到或低于 1 分与目前合并的过敏性结膜炎或基线血清包膜生长因子水平呈负相关。此外,信号检测分析表明,基线血清LDH水平低于328 U/L有可能被用作预测杜利单抗疗效的临界值。
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引用次数: 0
MASTering systemic mastocytosis: Lessons learned from a large patient cohort MASTering 系统性肥大细胞增多症:从大量患者中汲取的经验教训
Pub Date : 2024-07-27 DOI: 10.1016/j.jacig.2024.100316
Kevin Y. Tse MD, MS , Wansu Chen MS, PhD , Eric J. Puttock PhD , Shanta Chowdhury MS, PhD , Kerri Miller PharmD , Dakota Powell MPH , Benjamin Lampson MD, PhD , Chris Yuen PharmD , Doug Cattie PhD , Teresa Green MSPH , Erin Sullivan PhD, MPH , Robert S. Zeiger MD, PhD

Background

Systemic mastocytosis (SM), a rare condition affecting about 32,000 individuals in the United States, is often misdiagnosed or underdiagnosed owing to its nonspecific symptoms and the need for invasive biopsies.

Objective

Our aim was to identify, classify, and characterize the natural history of patients with SM.

Methods

In a retrospective cohort study, administrative data from a large managed care organization was used to identify patients with confirmed SM, based on World Health Organization criteria. Demographic data, delay to diagnosis, disease progression, and health care resource utilization were examined.

Results

Of 116 patients with confirmed SM, 77% had indolent SM, 2% had smoldering SM, 12% had SM with associated hematologic neoplasm, 9% had aggressive SM, and none had mast cell leukemia. In all, 5 patients were misclassified as having a less advanced SM subtype initially and 3 were completely undiagnosed (missed diagnosis). The average delay to diagnosis of SM was 58.3 plus or minus 73.1 months. In all, 18% of patients progressed from a nonadvanced form of SM (indolent or smoldering SM) to an advanced form of SM (aggressive SM, SM with associated hematologic neoplasm, or mast cell leukemia) over an average of 88.3 plus or minus 82.7 months. Patients with SM had increased health care utilization, including increases in their numbers of hospital admissions, emergency room visits, urgent care visits, and specialty provider visits, after diagnosis versus before.

Conclusions

Rare diseases such as SM would benefit from increased understanding and awareness to improve diagnostic accuracy. Prospective studies are needed to better characterize this patient population and determine the type of follow-up needed to recognize advanced forms of SM so that appropriate treatment can be implemented.

背景系统性肥大细胞增多症(SM)是一种罕见疾病,在美国约有 32,000 人患病,由于其症状无特异性,且需要进行侵入性活检,因此常常被误诊或漏诊。方法在一项回顾性队列研究中,我们根据世界卫生组织的标准,利用一家大型管理式医疗机构的管理数据来确定确诊的 SM 患者。结果 在116名确诊的肥大细胞白血病患者中,77%为惰性肥大细胞白血病,2%为烟雾型肥大细胞白血病,12%为伴有血液肿瘤的肥大细胞白血病,9%为侵袭性肥大细胞白血病,无肥大细胞白血病患者。共有5名患者最初被误诊为晚期肥大细胞白血病亚型,3名患者完全未确诊(漏诊)。肥大细胞白血病的平均诊断延迟时间为 58.3 个月正负 73.1 个月。总之,18%的患者在平均88.3个月正负82.7个月的时间内,从非晚期SM(惰性或烟熏型SM)发展为晚期SM(侵袭性SM、伴有血液肿瘤的SM或肥大细胞白血病)。肥大细胞白血病患者在确诊后与确诊前相比,使用医疗服务的次数增加,包括入院次数、急诊就诊次数、紧急护理就诊次数和专科医生就诊次数的增加。需要开展前瞻性研究,以更好地描述这类患者的特征,并确定识别晚期 SM 所需的随访类型,以便实施适当的治疗。
{"title":"MASTering systemic mastocytosis: Lessons learned from a large patient cohort","authors":"Kevin Y. Tse MD, MS ,&nbsp;Wansu Chen MS, PhD ,&nbsp;Eric J. Puttock PhD ,&nbsp;Shanta Chowdhury MS, PhD ,&nbsp;Kerri Miller PharmD ,&nbsp;Dakota Powell MPH ,&nbsp;Benjamin Lampson MD, PhD ,&nbsp;Chris Yuen PharmD ,&nbsp;Doug Cattie PhD ,&nbsp;Teresa Green MSPH ,&nbsp;Erin Sullivan PhD, MPH ,&nbsp;Robert S. Zeiger MD, PhD","doi":"10.1016/j.jacig.2024.100316","DOIUrl":"10.1016/j.jacig.2024.100316","url":null,"abstract":"<div><h3>Background</h3><p>Systemic mastocytosis (SM), a rare condition affecting about 32,000 individuals in the United States, is often misdiagnosed or underdiagnosed owing to its nonspecific symptoms and the need for invasive biopsies.</p></div><div><h3>Objective</h3><p>Our aim was to identify, classify, and characterize the natural history of patients with SM.</p></div><div><h3>Methods</h3><p>In a retrospective cohort study, administrative data from a large managed care organization was used to identify patients with confirmed SM, based on World Health Organization criteria. Demographic data, delay to diagnosis, disease progression, and health care resource utilization were examined.</p></div><div><h3>Results</h3><p>Of 116 patients with confirmed SM, 77% had indolent SM, 2% had smoldering SM, 12% had SM with associated hematologic neoplasm, 9% had aggressive SM, and none had mast cell leukemia. In all, 5 patients were misclassified as having a less advanced SM subtype initially and 3 were completely undiagnosed (missed diagnosis). The average delay to diagnosis of SM was 58.3 plus or minus 73.1 months. In all, 18% of patients progressed from a nonadvanced form of SM (indolent or smoldering SM) to an advanced form of SM (aggressive SM, SM with associated hematologic neoplasm, or mast cell leukemia) over an average of 88.3 plus or minus 82.7 months. Patients with SM had increased health care utilization, including increases in their numbers of hospital admissions, emergency room visits, urgent care visits, and specialty provider visits, after diagnosis versus before.</p></div><div><h3>Conclusions</h3><p>Rare diseases such as SM would benefit from increased understanding and awareness to improve diagnostic accuracy. Prospective studies are needed to better characterize this patient population and determine the type of follow-up needed to recognize advanced forms of SM so that appropriate treatment can be implemented.</p></div>","PeriodicalId":75041,"journal":{"name":"The journal of allergy and clinical immunology. Global","volume":"3 4","pages":"Article 100316"},"PeriodicalIF":0.0,"publicationDate":"2024-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772829324001127/pdfft?md5=0868b237a6118df8f57235816e80a64e&pid=1-s2.0-S2772829324001127-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141841274","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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The journal of allergy and clinical immunology. Global
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