Differences in Efficacy and Safety of Sirolimus and Sildenafil in Pediatric Lymphatic Malformations

Abstract

ObjectivesTo explore the differences in the efficacy and safety of oral sirolimus and sildenafil in the treatment of pediatric intractable lymphatic malformations (LMs).MethodsFrom January 2014 to May 2022, we retrospectively enrolled children with intractable LMs treated with oral drugs (sirolimus or sildenafil) and divided the patients into sirolimus and sildenafil groups from Beijing Children's Hospital (BCH). Clinical features, treatment, and follow‐up data were collected and analyzed. The indicators were the ratio of reduction in lesion volume pre and posttreatment, the number of patients with improved clinical symptoms, and adverse reactions to the two drugs.ResultsTwenty‐four children in the sildenafil group and 31 children in the sirolimus group were included in the present study. The effective rate in the sildenafil group was 54.2% (13/24), with a median lesion volume reduction ratio of 0.32 (−0.23, 0.89) and clinical symptoms improved in 19 patients (79.2%). On the contrary, the effective rate in the sirolimus group was 93.5% (29/31), with a median lesion volume reduction ratio of 0.68 (0.34, 0.96), and clinical symptoms improved in 30 patients (96.8%). There were significant differences (p < 0.05) between the two groups. Regarding safety, four patients in the sildenafil group and 23 patients in the sirolimus group with mild adverse reactions were reported.ConclusionBoth sildenafil and sirolimus can reduce the volume of LMs and improve clinical symptoms in partial patients with intractable LMs. Sirolimus is more effective than sildenafil and the adverse reactions associated with both drugs are mild and controllable.Level of EvidenceIII Laryngoscope, 2023

SMAC/Diablo controls proliferation of cancer cells by regulating phosphatidylethanolamine synthesis

“…Translocation of the protein from the nucleus to mitochondria induces apoptosis. (Nucleus, mitochondria) [78] 1L9-KADGIMWLAKA CWSIQSEMPCIQAQ 1M18-GVRTCEGCKGF FKRTVQKNAKYICL (Figs 7F and S7). Confocal images clearly show that the peptides were localized to the mitochondria or the nucleus, according to their targeting sequence.…”