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指定難病 — No.145

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検索語 West Syndrome ・ 最終更新 2026-07-21 20:11 ・ 最新に更新

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指定 No.145
Src PubMed · CT.gov · jRCT

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( 01 )EVIDENCE / PUBMED · 5件

世界の論文

直近の研究を、やさしい日本語で

各論文の見出しにある「確からしさ」は、その研究がどれくらい信頼できるかの目安です。「理論段階」はまだ仮説に近く、下にいくほど多くの患者で検証されていて、「メタ解析」がもっとも信頼できます。

観察研究
MK-01 · PMID 42479303

Epidemiology of Acute Colonic Pseudo-obstruction (Ogilvie Syndrome) Based on Disease-Specific Data

Abstract / 原文

PURPOSE: Prior epidemiologic assessment of acute colonic pseudo-obstruction (ACPO) used nonspecific ICD-9 coding preventing precise case identification. Implementation of disease-specific ICD-10-CM code K59.81 (Ogilvie syndrome) in 2020 enables more accurate characterization. METHODS: Adults with ICD-10-CM code K59.81 in the 2021-2022 National Inpatient Sample were identified. Patients were classified into four treatment groups: medical management, colonoscopy alone, surgery alone, and combined colonoscopy/surgery. Multivariable regression models assessed associations between treatment group and outcomes. RESULTS: Age/sex adjusted incidences of ACPO hospitalization were 3.99 and 4.03 per 100,000 population in 2021 and 2022. Incidence was higher in males than females (66% of cases were in men) and increased with age in both sexes. Medical management was the predominant treatment (77.9%), followed by colonoscopy alone (13.0%), surgery alone (6.6%), and combined colonoscopy/surgery (2.4%). Complication and mortality rates were 4.9% and 6.8%. In adjusted analyses, compared to medical management, surgery (OR = 11.48; 95% CI 7.45-17.71) and combined colonoscopy/surgery (OR = 10.41; 95% CI 5.49-19.73) had higher complications, surgery alone had higher mortality (OR = 2.51; 95% CI 1.87-3.37), colonoscopy alone trended toward lower mortality (OR = 0.67; 95% CI 0.45-1.00), and all procedural interventions had longer hospital stays. CONCLUSIONS: Disease-specific diagnostic coding for ACPO identified a population with pronounced male predominance and higher incidence in the elderly. Colonoscopy was used in 15% of cases and surgery in 9%. While most patients with ACPO require only medical management, colonoscopy was not associated with increased complications and potentially may be associated with reduced mortality. Surgery had the highest complication and mortality rates, likely reflecting greater disease severity in patients requiring operative intervention in addition to the risks of surgery.

Journal
Digestive diseases and sciences(2026 Jul)
Authors
2名
Type
Journal Article
PubMedで原文を見る
観察研究
MK-02 · PMID 42477577

The CALLY Index as an integrated inflammation-nutrition biomarker for risk stratification in HFpEF

Abstract / 原文

BACKGROUND: Heart failure with preserved ejection fraction (HFpEF) is a complex systemic syndrome characterized by inflammation, metabolic dysregulation, and immune imbalance. However, simple and integrative biomarkers for risk stratification remain limited. The C-reactive protein-albumin-lymphocyte (CALLY) index, reflecting inflammation, nutritional status, and immune function, has shown prognostic value in various diseases, but its role in HFpEF remains unclear. METHODS: In this single-center retrospective cohort study, 308 patients with HFpEF were enrolled. The CALLY index was calculated as albumin × lymphocyte count / CRP. The primary endpoint was major adverse cardiovascular events (MACE), defined as all-cause death or heart failure rehospitalization. Cox proportional hazards models, restricted cubic spline (RCS), receiver operating characteristic (ROC) analysis, and Kaplan-Meier curves were applied. RESULTS: During follow-up, 87 patients (28.25%) experienced MACE. Multivariable Cox analysis identified the CALLY index (HR = 0.98, 95% CI: 0.96-0.99) and E/e' (HR = 1.08, 95% CI: 1.01-1.17) as independent predictors of MACE. RCS analysis demonstrated a nonlinear inverse association between the CALLY index and MACE risk. ROC analysis showed good predictive performance (AUC = 0.82). Patients with lower CALLY index (< 11.8) had significantly higher event rates (log-rank p < 0.001). CONCLUSIONS: The CALLY index is an independent predictor of long-term outcomes in HFpEF, integrating inflammation, nutrition, and immune status. It may serve as a simple and practical tool for risk stratification.

Journal
BMC cardiovascular disorders(2026 Jul)
Authors
7名
Type
Journal Article
PubMedで原文を見る
非ランダム化試験
MK-03 · PMID 42476725

Phase 1/2 study of IMC-C103C, a T cell receptor bispecific (MAGE-A4×CD3) ImmTAC targeting MAGE-A4-expressing malignancies

Abstract / 原文

PURPOSE: IMC-C103C is an ImmTAC bispecific (MAGE-A4×CD3) T cell engager targeting a peptide from the cancer-testis antigen MAGE-A4 presented by HLA-A*02:01. This phase 1/2 study in advanced solid tumors (IMC-C103C-101; NCT03973333) investigated the safety and preliminary anti-tumor activity of IMC-C103C. METHODS: HLA-A*02:01+ patients with previously treated advanced solid tumors received IMC-C103C by weekly intravenous infusion, with step-up dosing introduced at higher dose levels. Dose-escalation decisions were guided by the mTPI-2 method. Key objectives included evaluating safety and preliminary anti-tumor activity; pharmacokinetics, pharmacodynamics, and biomarkers were also assessed. RESULTS: 68 patients were enrolled in 10 dose-escalation cohorts (n=56; 64% ovarian carcinoma (OC)) and one OC expansion cohort (n=12). In dose escalation, patients received doses ranging from 0.5 to 240 µg. The dose-limiting toxicity rate at the two highest dose levels (1/6 and 2/10) did not exceed the maximum tolerated dose. A lower-dose regimen of 15-45-140 µg, with clinical activity, and a more favorable tolerability profile, was selected as the expansion dose. There were no treatment-related discontinuations or deaths. The most common treatment-related adverse events were cytokine-mediated, including grade 1/2 cytokine release syndrome and associated symptoms. While MAGE-A4 expression was observed in most patient's tumors (71% positive), the level of expression was low (median H-score=16). Initial clinical and pharmacodynamic activity was observed at doses of 15 µg, with more consistent activity observed at doses ≥90 µg. At doses ≥90 µg, patients with MAGE-A4+ OC had deeper reductions in tumor size and circulating tumor DNA and trended to have longer overall survival than patients with MAGE-A4- OC. CONCLUSIONS: IMC-C103C was well tolerated at doses up to 140 µg, induced dose-dependent pharmacodynamic changes, and demonstrated clinical activity in multiple patients with MAGE-A4+ solid tumors. Clinical activity was dependent on MAGE-A4 expression; however, expression was especially low in the higher dosing cohorts, limiting adequate efficacy assessment. TRIAL REGISTRATION NUMBER: NCT03973333.

利益相反の可能性特許の出願人/保有者である記載あり/株式保有の記載あり
Journal
Journal for immunotherapy of cancer(2026 Jul)
Authors
20名
Type
Journal Article, Clinical Trial, Phase I, Clinical Trial, Phase II, Multicenter Study
PubMedで原文を見る
不明
MK-04 · PMID 42476512

COVID-19 and Global Perinatal Health: Lessons Learned on Maternal-Neonatal Outcomes, Infant Feeding Practices, and Healthcare Economics Across Income Settings

Abstract / 原文

BACKGROUND: The COVID-19 pandemic fundamentally disrupted healthcare systems worldwide, with disproportionate consequences for maternal and newborn health. Pregnant individuals and neonates faced both direct clinical consequences of acute respiratory syndrome - coronavirus-2 (SARS-CoV-2) infection and indirect effects of health system disruption. OBJECTIVE: This narrative review synthesizes global evidence on the pandemic's impact on perinatal outcomes, neonatal care, breastfeeding practices, and financial burden on healthcare institutions, contrasting high-income countries (HICs) and low- and middle-income countries (LMICs). DATA SOURCES: PubMed, the World Health Organization (WHO), the American Academy of Pediatrics (AAP), and the Centers for Disease Control and Prevention (CDC) databases were searched for peer-reviewed publications through early 2026. RESULTS: The pandemic increased maternal mortality, stillbirth, and neonatal morbidity, particularly in LMICs, alongside paradoxical preterm birth reductions in some HICs. Vertical SARS-CoV-2 transmission remained infrequent. Neonatal admissions fell while acuity and length of stay increased. Breastfeeding was profoundly disrupted by inconsistent separation policies and infection control measures. Healthcare institutions absorbed substantial additional costs from screening programs and care pathway reorganization. CONCLUSION: COVID-19 exposed and exacerbated existing global health inequities in maternal and neonatal care. Evidence-based, context-sensitive policies from WHO, AAP, and CDC are essential to preserve perinatal care quality during future health emergencies.

Journal
American journal of perinatology(2026 Jul)
Authors
5名
Type
Journal Article
PubMedで原文を見る
観察研究
MK-05 · PMID 42476001

Vigabatrin therapy for infantile epileptic spasms syndrome with periventricular leukomalacia

Abstract / 原文

BACKGROUND: Infantile epileptic spasm syndrome (IESS) and periventricular leukomalacia (PVL) cause developmental regression and poor neurological outcomes. The primary treatments for IESS include adrenocorticotropin and vigabatrin; however, no study has yet evaluated the efficacy of vigabatrin for patients with both IESS and PVL. Therefore, we investigated the efficacy and safety of vigabatrin in patients with both disorders. METHODS: We retrospectively enrolled patients with IESS and PVL who were treated with vigabatrin. We performed ophthalmological examinations and electroretinography (ERG) before and after vigabatrin. Treatment response was defined as complete cessation of epileptic spasms and no other seizures after 3 months of vigabatrin. We investigated the responses to vigabatrin, doses used, and adverse effects. RESULTS: Seven patients (5 males) with IESS and PVL were treated with vigabatrin. The median age at vigabatrin initiation was 10 (range, 7-25) months. Three of the seven patients (42.8%) showed complete clinical cessation of epileptic spasms after 3 months of vigabatrin. The median duration between vigabatrin initiation and epileptic spasm cessation was 5 (range: 3-61) days. The adverse effects included poor feeding (n = 1) and abnormal ERG findings during vigabatrin therapy (n = 2). These ERG findings normalized after vigabatrin was reduced or stopped. No respiratory failure, increased secretion, or somnolence was observed. CONCLUSION: Vigabatrin was effective for treating 42.8% of our patients with both IESS and PVL. Vigabatrin was well tolerated, with only a mild effect observed in patients with IESS and PVL. Some treatment-related ERG abnormalities were normalized after vigabatrin was reduced or stopped.

Journal
Brain & development(2026 Jul)
Authors
9名
Type
Journal Article
PubMedで原文を見る
( 02 )TRIALS / JAPAN · 0件

日本で参加できる治験

現在 募集中のもの

日本で現在募集中の治験は見つかりませんでした。下の公式レジストリで条件を変えると見つかる場合があります。
( 03 )REGISTRY / jRCT

治験をもっと探す

日本の公式レジストリで全件を確認

上の一覧は ClinicalTrials.gov の一部です。日本国内の治験の多くは、日本の公式レジストリ jRCT にのみ登録されています。下記から最新の全件をご確認ください。

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