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Fragility index analysis for randomized controlled trials of approved biologicals and small molecule drugs in inflammatory bowel diseases.
Bai, Xiaoyin; Wan, Ziqi; Li, Yi; Jiang, Qingwei; Wu, Xia; Xu, Bing; Li, Xiang; Zhou, Runing; Mi, Jiarui; Sun, Yinghao; Ruan, Gechong; Han, Wei; Li, Guanqiao; Yang, Hong.
Afiliación
  • Bai X; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
  • Wan Z; Eight-year Program, Peking Union Medical College and Chinese Academy of Medical Sciences, Beijing, China.
  • Li Y; Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China.
  • Jiang Q; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
  • Wu X; Department of Medicine, Tufts Medical Center, Boston, MA 02111, USA.
  • Xu B; Tsinghua University, China.
  • Li X; Tsinghua University, China.
  • Zhou R; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
  • Mi J; Department of Cell and Molecular Biology, Karolinska Institutet, Solna, Sweden.
  • Sun Y; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
  • Ruan G; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China.
  • Han W; Institute of Basic Medical Sciences, School of Basic Medicine, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, China.
  • Li G; Tsinghua University, China. Electronic address: guanqiaoli@tsinghua.edu.cn.
  • Yang H; Department of Gastroenterology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Beijing, China. Electronic address: yangh@pumch.cn.
Int Immunopharmacol ; 130: 111752, 2024 Mar 30.
Article en En | MEDLINE | ID: mdl-38422772
ABSTRACT

INTRODUCTION:

Biologics and small molecules have been increasingly applied in Crohn's disease (CD) and ulcerative colitis (UC). But the robustness of their trials has not been evaluated.

METHODS:

We initially collected all the approved biologics or small molecules for CD or UC up to December 1, 2022. Databases were then queried by keywords in chemical name and CD or UC. Randomized controlled trials (RCTs) in the two-arm, 11 design were included. Fragility index (FI) and fragility quotient (FQ) were subsequently calculated.

RESULTS:

We included twenty-eight RCTs, including nine pivotal trials listed in approval labels, nineteen non-pivotal trials not included in the labels. The median sample size was 99 [IQR, 60-262] and the median number of loss-of-follow-up (LFU) was 14 [IQR, 8-43]. Pivotal trials in the labels had the median FI of 8 [IQR, 4-14, n = 6] that was marginally higher than non-pivotal trials (3 [IQR, 2-4], p = 0.08). The median FQ was 0.0330 [IQR, 0.1220-0.0466] and 0.0310 [IQR, 0.0129-0.0540] for pivotal and non-pivotal trials, respectively (p = 1.0). The sample size and FI were significantly correlated (Spearman correlation coefficient [r] = 0.56, 95 %CI 0.21-0.78, p = 0.003). The number of total events was also significantly correlated with FI (r = 0.53, 95 %CI 0.17-0.77, p = 0.006). Study p-values were significantly associated with FI (p = 0.01) trials with p-values < 0.001 had the highest median FI of 10 [IQR, 6-17]. No factor was found strongly correlated with FQ.

CONCLUSION:

Results from trials assessing administration-approved biologics or small molecules for treating CD or UC were vulnerable to small changes by measuring FI or FQ. Pivotal studies contributing to regulatory approvals exhibited a relatively higher degree of resilience compared to non-pivotal trials.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Productos Biológicos / Enfermedades Inflamatorias del Intestino / Colitis Ulcerosa / Enfermedad de Crohn Límite: Humans Idioma: En Revista: Int Immunopharmacol Asunto de la revista: ALERGIA E IMUNOLOGIA / FARMACOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Países Bajos
Texto completo
Añadir a Mi BVS
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Productos Biológicos / Enfermedades Inflamatorias del Intestino / Colitis Ulcerosa / Enfermedad de Crohn Límite: Humans Idioma: En Revista: Int Immunopharmacol Asunto de la revista: ALERGIA E IMUNOLOGIA / FARMACOLOGIA Año: 2024 Tipo del documento: Article País de afiliación: China Pais de publicación: Países Bajos