Botulismitoksiini kliiniline kasutamine

Botulismitoksiin on tugev bioloogiline mürk, mille toime võib mürgistuse korral viia lõtvade lihashalvatuste ja hingamispuudulikkuse tõttu surmani. Botulismitoksiini seitsmest serotüübist kaht kasutatakse ravi eesmärkidel: esimest korda võeti botulismitoksiin kasutusele strabismi raviks, praegu on peamiseks kasutusnäidustuseks düstooniad ja spastilisus, aga viimastel aastatel on lisandunud mitmeid uusi näidustusi, nagu liigne süljevoolus, sulgurlihaste häired ja peavalud. Lisaks meditsiinilistele näidustusele on botulismitoksiini kasutatud ka kosmeetilistel eesmärkidel. Kuigi tegemist on tugevatoimelise mürgiga, on ravi kõrvaltoimeid suhteliselt vähe. Eestis alustati botulismitoksiini süstimist düstooniahaigetele 20 aastat tagasi, kuid praeguseks on näidustuste ring märkimisväärselt laienenud. Botulismitoksiini A-serotüübi preparaate (Botox ja Dysport) kasutatakse lisaks ka spastilisuse, liigse süljevooluse ja sulgurlihaste häirete raviks.Eesti Arst 2016; 95(3):171–17

Aju venoossete siinuste tromboos

Aju venoossed siinused on kõvakelme lestmete vahel moodustunud urked, mis koguvad verd suuraju veenidest ja suubuvad sisemisse kägiveeni. Venoossed siinused võivad tromboseeruda erinevatel lokaalsetel ja süsteemsetel põhjustel, kusjuures haigus võib avalduda äärmiselt heterogeense kliinilise sümptomaatikaga. Siinustromboosi diagnoosihüpotees vajab tõestust radioloogiliste uuringute abil ja õigeaegse diagnoosimise korral saab siinustromboosi edukalt ravida üle 90%-l juhtudest. Artiklis käsitletakse siinustromboosi kliinilist pilti, diagnoosimist ja ravi ning esitatakse andmed TÜ Kliinikumi neuroloogiaosakonnas ravitud juhtude kohta. Eesti Arst 2007; 86 (12): 897–90

A randomized, double-blind, placebo-controlled phase 2 study to assess safety, tolerability, and efficacy of RT001 in patients with amyotrophic lateral sclerosis

BACKGROUND AND PURPOSE: RT001 is a deuterated synthetic homologue of linoleic acid, which makes membrane polyunsaturated fatty acids resistant to lipid peroxidation, a process involved in motor neuron degeneration in amyotrophic lateral sclerosis (ALS). METHODS: We conducted a randomized, multicenter, placebo-controlled clinical trial. Patients with ALS were randomly allocated to receive either RT001 or placebo for 24 weeks. After the double-blind period, all patients received RT001 during an open-label phase for 24 weeks. The primary outcome measures were safety and tolerability. Key efficacy outcomes included the ALS Functional Rating Scale (ALSFRS-R), percent predicted slow vital capacity, and plasma neurofilament light chain concentration. RESULTS: In total, 43 patients (RT001 = 21; placebo = 22) were randomized. RT001 was well tolerated; one patient required dose reduction due to adverse events (AEs). Numerically, there were more AEs in the RT001 group compared to the placebo group (71% versus 55%, p = 0.35), with gastrointestinal symptoms being the most common (43% in RT001, 27% in placebo, p = 0.35). Two patients in the RT001 group experienced a serious AE, though unrelated to treatment. The least-squares mean difference in ALSFRS-R total score at week 24 of treatment was 1.90 (95% confidence interval = -1.39 to 5.19) in favor of RT001 (p = 0.25). The directions of other efficacy outcomes favored RT001 compared to placebo, although no inferential statistics were performed. CONCLUSIONS: Initial data indicate that RT001 is safe and well tolerated. Given the exploratory nature of the study, a larger clinical trial is required to evaluate its efficacy

Relationship between the MDS-UPDRS and Quality of Life:A large multicenter study of 3206 patients

BACKGROUND: The relationship between Health-Related Quality of Life (HRQoL) and MDS-UPDRS has not been fully studied so far. The aim of this study was to evaluate the relationship between all MDS-UPDRS components and HRQoL in a representative international cohort of PD patients. METHODS: We collected demographic and disease-related data as well as MDS-UPDRS and PDQ8 scales. Data were analyzed using correlations between PDQ8 and all MDS-UPDRS items, subsequently two hierarchical multiple regressions were performed, first between the scores of the MDS-UPDRS Parts and PDQ8 and second between individual items from those Parts demonstrating significant relationship to PDQ8 scores in the first regression. LASSO regression analyses were performed to evaluate the relationship between PDQ8 and all individual MDS-UPDRS items. RESULTS: A total of 3206 PD patients were included in the study. In the first regression analysis, PDQ8 was significantly related to MDS-UPDRS parts I and II, but not to III and IV. In the second regression model, significant contributions to PDQ8 were found for Part I items Fatigue, Pain, Depressed mood, Apathy; and Part II items Dressing, Doing hobbies, Freezing, Speech and Tremor. In the LASSO analysis, six Part I, seven Part II, three Part III and one Part IV items contributed to PDQ8 scores. The five items most significantly related to the model were Depressed mood, Dressing, Apathy, Pain and Fatigue. CONCLUSIONS: This is so far the largest study related to HRQoL issues in PD. Restrictions in activities of daily living and non-motor symptoms significantly contribute to HRQoL in PD