The aim of this paper was to describe the outcome of the therapeutic administration of allogenic mesenchymal stem cells obtained from Wharton’s jelly (WJ-MSCs) in children with cerebral palsy (CP) during a medical therapeutic experiment. We retrospectively analyzed the records of 109 patients recruited in daily clinical practice. Each patient received 1–10 injections and was examined by the same neurologist (study investigator (SI)) on the day of each infusion. The SI used a 6-point Likert scale to assess the quality of life (QoL) and self-sufficiency of the patients on the basis of the neurological examination. Children with >50% follow-ups after this administration were included into the quantitative analysis. In addition, the assessments of the parents and other health care professionals were obtained for 23 patients and compared with those of the SI. Forty-eight of 54 analyzed patients (88.9%) achieved some improvement in health status. Forty-eight (88.9%) patients experienced an increase in their QoL, and 21 patients (38.9%) achieved an increase in their self-sufficiency level. Improvement was achieved in 17 areas. Adverse events were mild and temporary except one case of epilepsy deterioration leading to treatment discontinuation. Age, body mass, and cell dose were not significant predictors of QoL response, contrary to epilepsy; developmental breakthrough was dose-dependent.

The aim of this narrative review is to report on the current knowledge regarding the clinical use of umbilical cord blood (CB) based on articles from PubMed and clinical trials registered on ClinicalTrials.gov. An increasing amount of evidence suggests that CB may be used for both early diagnostics and treatment of cerebral palsy. The acidity of CB and its biochemical parameters, including dozens of cytokines, growth factors, and other metabolites (such as amino acids, acylcarnitines, phosphatidylcholines, succinate, glycerol, 3-hydroxybutyrate, and O-phosphocholine) are predictors of future neurodevelopment. In addition, several clinical studies confirmed the safety and efficacy of CB administration in both autologous and allogeneic models, including a meta-analysis of five clinical trials involving a total of 328 participants. Currently, nine clinical trials assessing the use of autologous umbilical CB in children diagnosed with hypoxic-ischemic encephalopathy or cerebral palsy are in progress. The total population assessed in these trials exceeds 2500 patients.

Muscular dystrophies are genetically determined progressive diseases with no cause-related treatment and limited supportive treatment. Although stem cells cannot resolve the underlying genetic conditions, their wide-ranging therapeutic properties may ameliorate the consequences of the involved mutations (oxidative stress, inflammation, mitochondrial dysfunction, necrosis). In this study, we administered advanced therapy medicinal product containing umbilical cord-derived mesenchymal stem cells (UC-MSCs) to 22 patients with muscular dystrophies. Patients received one to five intravenous and/or intrathecal injections per treatment course in up to two courses every 2 months. Four standard doses of 10, 20, 30, or 40 × 106 UC-MSCs per injection were used; the approximate dose per kilogram was 1 × 106 UC-MSCs. Muscle strength was measured with a set of CQ Dynamometer computerized force meters (CQ Elektronik System, Czernica, Poland). Statistical analysis of muscle strength in the whole group showed significant improvement in the right upper limb (+4.0 N); left hip straightening (+4.5 N) and adduction (+0.5 N); right hip straightening (+1.0 N), bending (+7.5 N), and adduction (+2.5 N); right knee straightening (+8.5 N); left shoulder revocation (+13.0 N), straightening (+5.5 N), and bending (+6.5 N); right shoulder adduction (+3.0 N), revocation (+10.5 N), and bending (+5 N); and right elbow straightening (+9.5 N); all these differences were statistically significant. In six patients (27.3%) these changes led to improvement in gait analysis or movement scale result. Only one patient experienced transient headache and lower back pain after the last administration. In conclusion, UC-MSC therapy may be considered as a therapeutic option for these patients.

Hypoxic-ischaemic encephalopathy (HIE) is a severe complication of asphyxia at birth. Therapeutic hypothermia, the standard method for HIE prevention, is effective in only 50 of the cases. As the understanding of the immunological basis of these changes increases, experiments have begun with the use of cord blood (CB) because of its neuroprotective properties. Mechanisms for the neuroprotective effects of CB stem cells include antiapoptotic and anti-inflammatory actions, stimulation of angiogenesis, production of trophic factors, and mitochondrial donation. In several animal models of HIE, CB decreased oxidative stress, cell death markers, CD4+ T cell infiltration, and microglial activation; restored normal brain metabolic activity; promoted neurogenesis; improved myelination; and increased the proportion of mature oligodendrocytes, neuron numbers in the motor cortex and somatosensory cortex, and brain weight. These observations translate into motor strength, limb function, gait, and cognitive function and behaviour. In humans, the efficacy and safety of CB administration were reported in a few early clinical studies which confirmed the feasibility and safety of this intervention for up to 10 years. The results of these studies showed an improvement in the developmental outcomes over hypothermia. Two phase-2 clinical studies are ongoing under the United States regulations, namely one controlled study and one blinded study.

Wprowadzenie: Do czasu wybuchu epidemii SARS-CoV-2 usługi telemedyczne w zakresie opieki nad osobami żyjącymi z HIV w Polsce nie były objęte powszechnym ubezpieczeniem zdrowotnym. Dlatego doświadczenie w tej formie świadczenia opieki zdrowotnej jest niewielkie i wymagają oceny akceptacji przez pacjentów.Cel: Naszym celem było poznanie opinii osób żyjących z HIV na temat pierwszego powszechnego zastosowania telemedycyny w opiece nad osobami z HIV w Polsce.Materiał i metody: W badaniu stosowano ankiety składające się z modułu jakościowego (przeprowadzonego za pomocą wywiadów online przy użyciu techniki Computer-Assisted Web Interview) i modułu ilościowego (20 pytań, w tym pięć pytań otwartych i 15 pytań zamkniętych). W dystrybucji zaproszeń uczestniczyły cztery organizacje pozarządowe wspierające osoby żyjące z HIV w Polsce.Wyniki: 156 respondentów udzieliło odpowiedzi, 25% kobiet, 58% miało ponad 50 lat, 84% i 53% było heteroseksualnych. Większość respondentów została zdiagnozowana w publicznej placówce medycznej (51%) lub anonimowo (32%). 77/156 (49,3%) respondentów miało opinię na temat telemedycyny, z czego 22/77 (28,6%) odpowiedziało, że ich zdaniem telemedycyna ma przyszłość, a 29/77 (19%), że tak, ale pod pewnymi warunkami (stanowi to 66,2% osób mających opinię). 26/77 (33,8%) respondentów wskazało, że ich zdaniem telemedycyna nie ma przyszłości. Respondenci, którzy mieli negatywną opinię, częściej pochodzili z terenów wiejskich i mieli niższy poziom wykształcenia. W modelach regresji logistycznej posiadanie wykształcenia podstawowego zwiększało prawdopodobieństwo braku opinii ponad pięciokrotnie (OR = 5,37 [2,44 do 11,82], P< 0,0001). Czynnikami zmniejszającymi prawdopodobieństwo braku opinii były trudności w uzyskaniu wizyty (0,35 [0,15 do 0,81], P=0,01) i dostęp wyłącznie do telemedycyny (0,20 [0,06 do 0,72], P=0,01).Wnioski: Chociaż ponad 60% respondentów mających opinię wskazało potencjał telemedycyny w opiece nad osobami z HIV, połowa z nich wskazała konkretne warunki konieczne do jej wprowadzenia. Ocena leczenia HIV za pośrednictwem telemedycyny, a także akceptacja tej formy opieki przez pacjentów i dostawców musi być nadzorowana i korygowana w odniesieniu do zgłaszanych przez pacjentów miar wyników.