Epilepsy with myoclonic-atonic seizures (EMAS) is an early childhood epilepsy syndrome with an explosive onset of multiple seizure types in a previously healthy child. EMAS often evolves to a drug-resistant epileptic encephalopathy before a variable degree of remission around 3 years from onset. Highly purified cannabidiol (CBD, available in United States as Epidiolex®) has demonstrated efficacy and tolerability as an adjunct in medically refractory EMAS. We present two cases of medically refractory EMAS achieving rapid seizure freedom (7-30 days) with CBD, illustrating CBD as a potential effective monotherapy in EMAS.
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http://dx.doi.org/10.1002/epd2.20321 | DOI Listing |
BMC Rheumatol
January 2025
Department of Rheumatology, Overton Brooks VA Medical Center, Shreveport, LA, USA.
Background: Dermatomyositis is a chronic inflammatory condition affecting muscles and skin, often associated with an increased risk of cancer. Specific autoantibodies, including anti-TIF1 (Transcription Intermediary Factor 1), have been linked to this risk. We present a case of dermatomyositis in a male patient positive for anti-TIF1 antibodies, subsequently diagnosed with squamous cell carcinoma of the tonsil, a novel association not previously documented.
View Article and Find Full Text PDFClin Lymphoma Myeloma Leuk
January 2025
Transplantation & Cellular Therapy Program, Division of Hematology/Oncology, Department of Internal Medicine, Greenebaum Comprehensive Cancer Center, University of Maryland Medical Center, University of Maryland School of Medicine, Baltimore, MD.
Background: Anti-CD19 CAR-T therapy has been a breakthrough in treatment of primary refractory or relapsed large B-cell lymphoma (r/r LBCL) and is poised to supplant previous second line of high dose chemotherapy and autologous stem cell transplantation (HDT/ASCT). However, in clinical practice, high risk patients with chemoimmunotherapy sensitive disease continue to receive salvage chemoimmunotherapy or cannot access CAR-T in a timely manner and thus may still proceed to HDT/ASCT. Little is known about clinical outcomes of CAR-T in patients who receive HDT/ASCT compared to those who are transplant-naïve.
View Article and Find Full Text PDFVitam Horm
January 2025
Department of Physiology, School of Medicine, Tehran University of Medical Sciences, Tehran, Iran. Electronic address:
Opioid use disorder (OUD) is considered a global health issue that affects various aspects of patients' lives and poses a considerable burden on society. Due to the high prevalence of remissions and relapses, novel therapeutic approaches are required to manage OUD. Deep brain stimulation (DBS) is one of the most promising clinical breakthroughs in translational neuroscience.
View Article and Find Full Text PDFDiscov Oncol
January 2025
Division of Hematology/Oncology, The University of Texas Health Sciences Center at Houston, McGovern Medical School, 6431 Fannin Street, MSB 5.216, Houston, TX, 77030, USA.
The established protocol for the management of acute myeloid leukemia (AML) has traditionally involved the administration of induction chemotherapy, followed by consolidation chemotherapy, and subsequent allogeneic stem cell transplantation for eligible patients. However, the prognosis for individuals with relapsed and refractory AML remains unfavorable. In response to the necessity for more efficacious therapeutic modalities, targeted immunotherapy has emerged as a promising advancement in AML treatment.
View Article and Find Full Text PDFCytotherapy
January 2025
Osteoarthritis Research Program, Division of Orthopedic Surgery, Schroeder Arthritis Institute, University Health Network, Toronto, Ontario, Canada; Krembil Research Institute, University Health Network, Toronto, Ontario, Canada; Institute of Biomedical Engineering, University of Toronto, Toronto, Ontario, Canada; Department of Medicine, Division of Hematology, University of Toronto, Toronto, Ontario, Canada. Electronic address:
The December 2024 US Food and Drug Administration (FDA) approval of Mesoblast's Ryoncil (remestemcel-L-rknd)-allogeneic bone marrow mesenchymal stromal cell (MSC(M)) therapy-in pediatric acute steroid-refractory graft-versus-host-disease finally ended a long-lasting drought on approved MSC clinical products in the United States. While other jurisdictions-including Europe, Japan, India, and South Korea-have marketed autologous or allogeneic MSC products, the United States has lagged in its approval. The sponsor's significant efforts and investments, working closely with the FDA addressing concerns regarding clinical efficacy and consistent MSC potency through an iterative process that spanned several years, was rewarded with this landmark approval.
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