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and revision rotator cuff tears led to improved functional outcomes. Approximately half of patients experienced a failure of healing, which was associated with poorer functional results.We examined safety, tolerability, and efficacy of SGS-742, a γ-aminobutyric acid B (GABA-B) receptor antagonist, in patients with succinic semialdehyde dehydrogenase deficiency. This was a single-center randomized, double-blind crossover phase II clinical trial of SGS-742 versus placebo in patients with succinic semialdehyde dehydrogenase deficiency. Procedures included transcranial magnetic stimulation and the Adaptive Behavior Assessment Scale. Nineteen subjects were consented and enrolled; the mean age was 14.0 ± 7.5 years and 11 (58%) were female. We did not find a significant effect of SGS-742 on the Adaptive Behavior Assessment Scale score, motor threshold, and paired-pulse stimulation. The difference in recruitment curve slopes between treatment groups was 0.003 (P = .09). There was no significant difference in incidence of adverse effects between drug and placebo arms. SGS-742 failed to produce improved cognition and normalization of cortical excitability as measured by the Adaptive Behavior Assessment Scale and transcranial magnetic stimulation. Our data do not support the current use of SGS-742 in succinic semialdehyde dehydrogenase deficiency.Trial registry number NCT02019667. selleck kinase inhibitor Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency. https//clinicaltrials.gov/ct2/show/NCT02019667.
The ongoing SARS-CoV-2 pandemic is a serious threat for the health of immunocompromised patients. Among neutralizing antibody-based therapeutics, convalescent plasma containing polyclonal anti-SARS-CoV-2 immunoglobulins has promising results in both congenital and iatrogenic immunodeficiencies in oncohematological and transplant patients.
This article discusses case reports, case series and controlled studies detailing the efficacy of convalescent plasma in immunocompromised patients.
Convalescent plasma, when administered at high neutralizing antibody titers, is a safe and effective treatment for frail immunocompromised patients. Genetic monitoring of refractory patients is recommended to intercept intra-host emergence of SARS-CoV-2 variants.
Convalescent plasma, when administered at high neutralizing antibody titers, is a safe and effective treatment for frail immunocompromised patients. Genetic monitoring of refractory patients is recommended to intercept intra-host emergence of SARS-CoV-2 variants.
Patients with idiopathic pulmonary fibrosis (IPF) frequently suffer from difficult to treat chronic cough, which substantially affects their quality of life. Azithromycin has been demonstrated to relieve chronic cough in some populations, however this has not been investigated in IPF.
To determine the safety and efficacy of azithromycin for the treatment of chronic cough in patients with IPF.
In a double-blind randomized controlled cross-over trial, patients with IPF underwent two 12-week intervention periods (azithromycin 500mg or placebo 3 times per week). The primary outcome was change in cough-related quality of life measured by the Leicester cough questionnaire (LCQ). Secondary outcomes included cough severity measured using Visual Analog Scale (VAS), health-related quality of life assessed by the St. George's Respiratory Questionnaire (SGRQ), and objective cough frequency using audiovisual readings from 24h respiratory polygraphy.
25 patients were randomized (23 men, 2 women), 20 patients comple registered with ClinicalTrials.gov (NCT02173145).With the progress of COVID-19 vaccination programs worldwide, some new adverse events associated with the available vaccines may unfold, especially in subpopulations, representatives of whom were not included in phase I, II, and III clinical trials of these vaccines, such as patients with autoimmune diseases, including multiple sclerosis (MS). A 34-year-old woman presented with severe right hemiplegia and ataxia. She was diagnosed with relapsing-remitting MS (RRMS) 13 years ago and treated with rituximab (an anti-CD20 monoclonal antibody) during the last 15 months. She had received her first dose of adenovirus-vectored COVID-19 vaccine Gam-COVID-Vac (Sputnik V) three months after her last infusion of rituximab and three days before experiencing her latest MS relapse episode, preceded by mild symptoms (fatigue, myalgia, generalized weakness, etc.). Magnetic resonance imaging revealed several new periventricular, juxtacortical, brainstem, and cerebellar peduncle lesions. She received corticosteroid therapy for five consecutive days, and her neurological deficits slightly improved. Twenty-one days after receiving the first dose of the vaccine, her anti-SARS-CoV-2 antibodies were below the lower detection limit. However, a decision was made to adhere to the vaccination schedule and not risk the patient's safety against an unfortunate COVID-19 contraction, and thus, she was advised to receive the second Gam-COVID-Vac dose after discontinuation of oral steroid taper. The safety of adenovirus-based vaccines in patients with autoimmune diseases requires further investigation. Meanwhile, clinicians should raise awareness among their patients regarding the potentially limited efficacy of COVID-19 vaccination in those treated with anti-CD20 treatments. After careful, individualized risk-benefit assessments, planning a delay/pause in such treatments to create a time window for patients to receive the vaccine and develop anti-SARS-CoV-2 immunity may be recommended.
Recurrent tears after arthroscopic rotator cuff repair (ARCR) remain a significant clinical problem. Oxidative stress contributes to the degeneration of the rotator cuff, and a degenerative rotator cuff can lead to recurrent tear after ARCR. However, the correlation between oxidative stress and retear after ARCR is unclear.
To investigate the correlation between superoxide-induced oxidative stress and recurrent tear after ARCR.
Case-control study; Level of evidence, 3.
A total of 68 patients who underwent ARCR using a suture-bridge technique participated in this study. Specimens were collected from the edge of the torn tendon during surgery. The modified Bonar score was used to evaluate degeneration of the rotator cuff on histological specimens, and fluorescence intensity on dihydroethidium (DHE) staining was used to detect oxidative stress. Superoxide dismutase (SOD) enzyme activity was also measured. The following were used for clinical evaluation age, tear size on magnetic resonance imaging (MRI) before surgery, Goutallier classification on MRI before surgery, and Japanese Orthopaedic Association score before and 6 months after surgery.
