Objective Amyotrophic lateral sclerosis (ALS) is still incurable. Although different therapies can affect the health and survival of patients. Our aim is to evaluate the effect of umbilical mesenchymal stem cells administrated intrathecally to patients with amyotrophic lateral sclerosis on disability development and survival. Methods This case-control study involved 67 patients treated with Wharton’s jelly mesenchymal stem cells (WJ-MSC). The treated patients were paired with 67 reference patients from the PRO-ACT database which contains patient records from 23 ALS clinical studies (phase 2/3). Patients in the treatment and reference groups were fully matched in terms of race, sex, onset of symptoms (bulbar/spinal), FT9 disease stage at the beginning of therapy and concomitant amyotrophic lateral sclerosis medications. Progression rates prior to treatment varied within a range of ± 0.5 points. All patients received three intrathecal injections of Wharton’s jelly-derived mesenchymal stem cells every two months at a dose of 30 × 10 6 cells. Patients were assessed using the ALSFRS-R scale. Survival times were followed-up until March 2020. Results Median survival time increased two-fold in all groups. In terms of progression, three response types measured in ALSFRS-R were observed: decreased progression rate ( n = 21, 31.3%), no change in progression rate ( n = 33, 49.3%) and increased progression rate ( n = 13, 19.4%). Risk-benefit ratios were favorable in all groups. No serious adverse drug reactions were observed. Interpretation Wharton’s jelly-derived mesenchymal stem cells therapy is safe and effective in some ALS patients, regardless of the clinical features and demographic factors excluding sex. The female sex and a good therapeutic response to the first administration are significant predictors of efficacy following further administrations. Graphical Abstract Medical therapeutic experiment with retrospective case-control analyses Electronic supplementary material The online version of this article (10.1007/s12015-020-10016-7) contains supplementary material, which is available to authorized users.
Amyotrophic lateral sclerosis (ALS) is a rare, incurable, and fatal neurodegenerative disease with median survival time from onset to death ranging from 20 to 48 months. Breaking bad news about ALS diagnosis is a challenging task for physicians and a life-changing experience for patients. Several protocols for delivering difficult information are available, including SPIKES and EMPATHY. Our goal was to assess to what extent these guidelines are followed in Polish ALS patients’ experience as well as to identify any other patients’ preferences not addressed by the guidelines. Participants of our study were recruited via a neurology clinic. Twenty-four patients with confirmed ALS diagnosis were interviewed using in-depth interview and a self-constructed questionnaire: 9 females, 15 males in age ranging from 30–39 to 60–69. The analysis showed a pattern of shortcomings and fundamental violations of available protocols reported by ALS patients. Patients also had to deal with therapeutic nihilism, as they were perceived as “hopeless cases”; unlike in oncological setting, their end-of-life needs were not accommodated by some standard schemes. As a conclusion, we recommend using extended breaking bad news protocols with special emphasis on preparing a treatment plan, giving the patient hope and sense of purpose, offering psychological support and counselling directed to patients and caregivers, and providing the patient with meaningful information about the disease, social support, treatment options, and referral to appropriate health care centres.
Animal experiments have confirmed that mesenchymal stem cells can inhibit motor neuron apoptosis and inflammatory factor expression and increase neurotrophic factor expression. Therefore, mesenchymal stem cells have been shown to exhibit prospects in the treatment of amyotrophic lateral sclerosis. However, the safety of their clinical application needs to be validated. To investigate the safety of intrathecal injection of Wharton’s jelly-derived mesenchymal stem cells in amyotrophic lateral sclerosis therapy, 43 patients (16 females and 27 males, mean age of 57.3 years) received an average dose of 0.42 × 106 cells/kg through intrathecal administration at the cervical, thoracic or lumbar region depending on the clinical symptoms. There was a 2 month interval between two injections. The adverse events occurring during a 6-month treatment period were evaluated. No adverse events occurred. Headache occurred in one case only after first injection of stem cells. This suggests that intrathecal injection of Wharton’s Jelly-derived mesenchymal stem cells is well tolerated in patients with amyotrophic lateral sclerosis. This study was approved by the Bioethical Committee of School of Medicine, University of Warmia and Mazury in Olsztyn, Poland (approval No. 36/2014 and approval No. 8/2016). This study was registered with the ClinicalTrials.gov (identifier: NCT02881476) on August 29, 2016.
The COVID-19 pandemic has revealed the high usefulness of telemedicine. To date, no uniform recommendations or diagnostic protocols for long-COVID patients have been developed. This article presents the preliminary results of the examination of patients after SARS-CoV-2 infection who were provided with medical telemonitoring devices in order to oversee their pulmonological and cardiological health. Three cases have been analyzed. Each patient underwent a 10-day registration of basic vital signs, in three 15-min sessions daily: RR (respiratory rate), ECG (electrocardiogram), HR (pulse), SPO2 (saturation), body temperature and cough. Rule methods and machine learning were employed to automatically detect events. As a result, serious disorders of all the three patients were detected: cardiological and respiratory disorders that required extended diagnostics. Furthermore, average values of the selected parameters (RR, HR, SPO2) were calculated for every patient, including an indication of how often they exceeded the alarm thresholds. In conclusion, monitoring parameters in patients using telemedicine, especially in a time of limited access to the healthcare system, is a valuable clinical instrument. It enables medical professionals to recognize conditions which may endanger a patient’s health or life. Telemedicine provides a reliable assessment of a patient’s health status made over a distance, which can alleviate a patient’s stress caused by long-COVID syndrome. Telemedicine allows identification of disorders and performing further diagnosis, which is possible owing to the implementation of advanced analysis. Telemedicine, however, requires flexibility and the engagement of a multidisciplinary team, who will respond to patients’ problems on an ongoing basis.
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