Linaclotide for the treatment of refractory lower bowel manifestations of systemic sclerosis.
Linaclotide for the treatment of refractory lower bowel manifestations of systemic sclerosis.
复制标题
利那洛肽治疗系统性硬化症的难治性下肠道表现。
DOI：
10.1186/s12876-021-01738-0
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发表时间：
2021-04-15
影响因子：
2.4
通讯作者：
McMahan ZH
中科院分区：
文献类型：
--
作者：
Dein EJ;Wigley FM;McMahan ZH
下消化道（GI）受累可影响高达50%的系统性硬化症（SSc）患者，并可能导致吸收不良、假性梗阻、住院和死亡。我们报告了利那洛肽（鸟苷酸环化酶C（GC-C）的选择性激动剂）治疗难治性下消化道疾病的SSc患者的经验。我们对约翰霍普金斯硬皮病中心的患者进行了分析，并确定了使用利那洛肽治疗难治性下消化道表现的患者。患者的临床数据收集在我们的纵向数据库中。利那洛肽应答者接受药物治疗至少12个月，治疗医生记录了有效性。31例SSc患者接受利那洛肽治疗。在开始利那洛肽治疗时，这些患者中有23例（74%）被归类为重度GI疾病，定义为复发性假性梗阻、吸收不良和/或需要人工营养（Medsger GI严重程度评分≥ 3）。大多数患者（90.3%; 28/31）有治疗反应，而只有3例患者（9.7%）报告无效或不可耐受的副作用。18例患者使用低剂量利那洛肽（每日≤ 145 mcg），有效率为94%。高剂量治疗（> 145 mcg/天）对13例患者中的11例（85%）有效。常见的副作用是腹泻、痉挛或腹胀（11/31，35%）。无效、费用和腹痛是那些停止治疗的人的抱怨。利那洛肽是一种耐受性良好且有效的促分泌和促运动剂，可用于治疗SSc的难治性下GI表现。我们发现，低剂量利那洛肽是一种有效的选择，可能会更好地耐受，虽然一个子集的患者可能需要高剂量的方案。
Lower gastrointestinal (GI) tract involvement can affect up to 50% of systemic sclerosis (SSc) patients, and may result in malabsorption, pseudo-obstruction, hospitalization, and death. We report our experience with linaclotide, a selective agonist of guanylate cyclase C (GC-C), for SSc patients with refractory lower GI disease. We performed an analysis of patients seen at the Johns Hopkins Scleroderma Center and identified patients prescribed linaclotide for refractory lower GI manifestations. Patients had clinical data collected in our longitudinal database. Linaclotide responders were on medication for at least 12 months with documented effectiveness by the treating physician. Thirty-one patients with SSc were treated with linaclotide. At the time of linaclotide initiation, 23 of these patients (74%) were classified as having severe GI disease, as defined by recurrent pseudo-obstruction, malabsorption, and/or need for artificial nutrition (Medsger GI severity score ≥ 3). The majority of patients (90.3%; 28/31) had a treatment response, while only three patients (9.7%) reported ineffectiveness or intolerable side effects. Low-dose linaclotide (≤ 145 mcg daily) was used in 18 patients and was effective in 94%. High-dose therapy (> 145 mcg daily) was effective in 11 of 13 patients (85%). Common side effects were diarrhea, cramping, or bloating (11/31, 35%). Ineffectiveness, cost, and abdominal pain were complaints cited among those who discontinued therapy. Linaclotide is a well-tolerated and efficacious pro-secretory and pro-motility agent that can be used to manage refractory lower GI manifestations in SSc. We found that low-dose linaclotide is an effective option and may be better tolerated, though a subset of patients may require high dose regimens.
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影响因子：
--
作者：
van den Hoogen, Frank;Khanna, Dinesh;Fransen, Jaap;Johnson, Sindhu R.;Baron, Murray;Tyndall, Alan;Matucci-Cerinic, Marco;Naden, Raymond P.;Medsger, Thomas A., Jr.;Carreira, Patricia E.;Riemekasten, Gabriela;Clements, Philip J.;Denton, Christopher P.;Distler, Oliver;Allanore, Yannick;Furst, Daniel E.;Gabrielli, Armando;Mayes, Maureen D.;van Laar, Jacob M.;Seibold, James R.;Czirjak, Laszlo;Steen, Virginia D.;Inanc, Murat;Kowal-Bielecka, Otylia;Mueller-Ladner, Ulf;Valentini, Gabriele;Veale, Douglas J.;Vonk, Madelon C.;Walker, Ulrich A.;Chung, Lorinda;Collier, David H.;Csuka, Mary Ellen;Fessler, Barri J.;Guiducci, Serena;Herrick, Ariane;Hsu, Vivien M.;Jimenez, Sergio;Kahaleh, Bashar;Merkel, Peter A.;Sierakowski, Stanislav;Silver, Richard M.;Simms, Robert W.;Varga, John;Pope, Janet E.
通讯作者：
Pope, Janet E.
影响因子：
9.8
作者：
Chey, William D.;Lembo, Anthony J.;Johnston, Jeffrey M.
通讯作者：
Johnston, Jeffrey M.
影响因子：
5
作者：
Gyger, Genevieve;Baron, Murray
通讯作者：
Baron, Murray
影响因子：
4
作者：
Brierley, Stuart M.
通讯作者：
Brierley, Stuart M.
影响因子：
4.6
作者：
Cao H;Liu X;An Y;Zhou G;Liu Y;Xu M;Dong W;Wang S;Yan F;Jiang K;Wang B
通讯作者：
Wang B