Efikasnost i sigurnost mTOR inhibitora (rapamicin i njegovi analozi) za kompleks tuberozne skleroze: meta-analiza.
Orphanet journal of rare diseases
Sažetak istraživanja
Pozadina Liječenje kompleksa tuberozne skleroze (TSC) korištenjem inhibitora rapamicina (mTOR) kod sisara je klinički obećavajuće.
Cilj ove studije bio je procijeniti efikasnost i sigurnost mTOR inhibitora za poboljšanje kliničkih simptoma TSC.
Metode Izvršili smo sistematsko pretraživanje glavnih elektronskih baza podataka (PubMed, EMBASE, Cochrane Library i WanFang, CNKI i VIP baze podataka) da bismo identifikovali randomizirana kontrolirana ispitivanja (RCT) i kvazi-randomizirane studije od datuma početka baze podataka do novembra 2017.; Kineska uprava za hranu i lijekove i Clinictrials.gov su također pretraženi za neobjavljene studije. Krajnje tačke studije bile su stopa odgovora tumora i stopa odgovora učestalosti napadaja (udio pacijenata koji su postigli smanjenje od ≥ 50% u odnosu na početnu liniju). Dva istraživača su pregledala članke, procijenila rizik od pristrasnosti i nezavisno izdvojila podatke. Uključeni RCT-ovi su analizirani pomoću RevMan-a 5.3, koji je obezbijedila Cochrane Collaboration.
Rezultati U poređenju sa placebom, mTOR inhibitori su značajno smanjili volumen tumora i kod angiomiolipoma (AML) (RR = 24,69, 95% CI = 3,51,173,41, P = 0,001) i kod subependimalnog astrocitoma džinovskih ćelija (SEGA) (RR5, 295% CI). 1,74,444,82, P = 0,02). U poređenju sa placebom, mTOR inhibitori su značajno smanjili učestalost napadaja (RR = 2,12, 95% CI = 1,41, 3,19, P = 0,0003). Što se tiče sigurnosti, u poređenju sa pacijentima koji nisu primali mTOR inhibitore, oni koji jesu imali su veći rizik od stomatitisa (RR = 3,20, 95% CI = 1,49, 6,86, P = 0,003). Nasuprot tome, pacijenti koji su primali ili nisu primali mTOR inhibitore imali su slične nuspojave, kao što su infekcije gornjih disajnih puteva (RR = 1,08, 95% CI = 0,81,1,45, P = 0,59) i nazofaringitis (RR = 0,86, 95% CI = 0,61,8,1).
Zaključak S obzirom na efikasnost i sigurnost povezane s tumorom i učestalošću napadaja kod pacijenata sa TSC, mTOR inhibitori su dobar terapijski izbor. Za razliku od rizika od infekcija gornjih disajnih puteva i nazofaringitisa, čini se da mTOR inhibitori povećavaju rizik od stomatitisa, uglavnom stepena 1 i 2.
Prikaži originalni naslov i sažetak na engleskom
Efficacy and safety of mTOR inhibitors (rapamycin and its analogues) for tuberous sclerosis complex: a meta-analysis.
Background The treatment of tuberous sclerosis complex (TSC) using mammalian target of rapamycin (mTOR) inhibitors is clinically promising. The aim of the present study was to evaluate the efficacy and safety of mTOR inhibitors for improving the clinical symptoms of TSC.
Methods We performed a systematic search of major electronic databases (PubMed, EMBASE, Cochrane Library and WanFang, CNKI, and VIP databases) to identify randomized controlled trials (RCTs) and quasi-randomized studies from the date of database inception to November 2017; the Chinese Food and Drug Administration and clinicaltrials.gov were also searched for unpublished studies. The endpoints of the study were the tumor response rate and seizure frequency response rate (the proportion of patients achieving a ≥ 50% reduction relative to the baseline). Two researchers screened articles, assessed the risk of bias and extracted data independently. The included RCTs were analyzed using RevMan 5.3, which was provided by the Cochrane Collaboration.
Results Compared with the placebo, mTOR inhibitors significantly reduced tumor volume in both angiomyolipoma (AML) (RR = 24.69, 95% CI = 3.51,173.41, P = 0.001) and subependymal giant cell astrocytoma (SEGA) (RR = 27.85, 95% CI = 1.74,444.82, P = 0.02). Compared with the placebo, mTOR inhibitors significantly reduced seizure frequency (RR = 2.12, 95% CI = 1.41,3.19, P = 0.0003). Regarding safety, compared with patients who did not receive mTOR inhibitors, those who did had a higher risk of suffering stomatitis (RR = 3.20, 95% CI = 1.49,6.86, P = 0.003). In contrast, patients who did and did not receive mTOR inhibitors experienced similar adverse events, such as upper respiratory tract infections (RR = 1.08, 95% CI = 0.81,1.45, P = 0.59) and nasopharyngitis (RR = 0.86, 95% CI = 0.60,1.21, P = 0.38).
Conclusion In view of the efficacy and safety associated with tumor and seizure frequency in the TSC patients, mTOR inhibitors is a good therapeutic choice. Unlike the risks of upper respiratory tract infections and nasopharyngitis, mTOR inhibitors seem to increase the risk of stomatitis, mostly grade 1 and 2.
Izvorni podaci
- DOI
- 10.1186/s13023-019-1012-x
- PMID
- 30760308
- PMCID
- PMC6373010
- Provjereno
- 2026-07-26
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