Vascular Biology Program, Department of Surgery, Boston Children's Hospital, Boston, Massachusetts, USA.
Harvard Medical School, Boston, Massachusetts, USA.
JPEN J Parenter Enteral Nutr. 2023 Nov;47(8):1028-1037. doi: 10.1002/jpen.2563. Epub 2023 Oct 8.
Enteral drug therapy is challenging in short bowel syndrome with intestinal failure (SBS-IF) because of unpredictable absorption. SEFA-6179 is an enterally administered medium-chain fatty acid analogue under development for intestinal failure-associated liver disease. We investigate the pharmacokinetics of two SEFA-6179 formulations in two large-animal models of SBS-IF, including a new pseudojejunostomy model.
Twenty Yucatan minipigs were obtained. Half underwent pre-resection pharmacokinetic study with single-dose SEFA-6179 administration. All minipigs then underwent 90% jejunoileal resection, with either a jejunoileal anastomosis or bypass of the intraperitoneal colon with anastomosis just proximal to the rectum (pseudojejunostomy). On postoperative day 3, a single-dose pharmacokinetic study was performed.
Both SBS-IF models were well tolerated. Compared with the jejunoileal anastomosis minipigs, pseudojejunostomy minipigs had a more severe malabsorptive phenotype with weight loss by postoperative day 4 (+0.1 vs -0.9 kg, P = 0.03) and liquid diarrhea (Bristol 5 vs Bristol 7, P = 0.0007). Compared with pre-resection minipigs, both jejunoileal and pseudojejunostomy minipigs had lower total plasma exposure of SEFA-6179 measured by area under the curve (jejunoileal: 37% less, P = 0.049; pseudojejunostomy: 74% less, P = 0.0001). Peak plasma concentration was also lower in the pseudojejunostomy group compared with pre-resection (65% less, P = 0.04), but not lower in the jejunoileal group (P = 0.47).
In two SBS-IF minipig models, SEFA-6179 had substantially decreased absorption compared with pre-resection minipigs. Dose optimization for different intestinal anatomy and function may be required. We describe a new SBS-IF pseudojejunostomy model that may improve the translation of preclinical research to patients with SBS-IF who have enterostomies.
由于吸收不可预测,短肠综合征伴肠衰竭(SBS-IF)患者的肠内药物治疗极具挑战性。SEFA-6179 是一种正在开发用于治疗与肠衰竭相关肝病的肠内给予的中链脂肪酸类似物。我们在两种 SBS-IF 大型动物模型中研究了两种 SEFA-6179 制剂的药代动力学,包括一种新的假性空肠造口术模型。
获得 20 只尤卡坦小型猪。其中一半进行了单剂量 SEFA-6179 给药的术前药代动力学研究。所有小型猪随后接受 90%空回肠切除术,其中一半进行空回肠吻合术,另一半进行腹腔内结肠旁路吻合术(位于直肠近端的假性空肠造口术)。术后第 3 天,进行单次剂量药代动力学研究。
两种 SBS-IF 模型均耐受良好。与空回肠吻合术小型猪相比,假性空肠造口术小型猪表现出更严重的吸收不良表型,术后第 4 天体重减轻(0.1 比-0.9kg,P=0.03),出现液体腹泻(Bristol 5 比 Bristol 7,P=0.0007)。与术前小型猪相比,空回肠和假性空肠造口术小型猪的 SEFA-6179 总血浆暴露量均较低(空回肠:少 37%,P=0.049;假性空肠造口术:少 74%,P=0.0001)。假性空肠造口术组的血浆峰浓度也低于术前(少 65%,P=0.04),而空回肠组则没有(P=0.47)。
在两种 SBS-IF 小型猪模型中,SEFA-6179 的吸收与术前小型猪相比明显降低。可能需要根据不同的肠道解剖和功能进行剂量优化。我们描述了一种新的 SBS-IF 假性空肠造口术模型,该模型可能有助于将临床前研究转化为患有肠造口术的 SBS-IF 患者。