Think Stop before Going 'Off-Label' by Gerald Y Minuk

“…Only after careful and extensive review of all available safety and efficacy data derived from preclinical and clinical trials will a regulatory body approve the licensing of a drug for a specific medical condition ('indication'). Experience with thalidomide, fialuridine and troglitazone, to name a few, reveal that despite these efforts, the process is not infallible. …”
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Management of Fistulas in Patients with Crohn'S Disease: Antibiotic to Antibody by Pierre Paré

“…Antibiotics, cyclosporine, methotrexate and thalidomide have been used in uncontrolled trials; only azathioprine, 6-mercaptopurine and infliximab have been assessed in double-blind, placebo controlled studies. …”
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Case of Stroke from Cerebral Vasculitis following Carfilzomib, Lenalidomide, and Dexamethasone Therapy in a Patient with Relapsing Multiple Myeloma by Deborah Osafehinti, Kaveh Zivari

“…Lenalidomide, a synthetic derivation of thalidomide, in recent years, has been the backbone of multiple myeloma treatment leading to improved survival. …”
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Isolated Langerhans Cell Histiocytosis of the Vulva in a 28-Year-Old Lady: A Report of a Case and Brief Review of the Literature by Maryam Sadat Sadati, Nafiseh Todarbary, Fatemeh Sari Aslani, Maryam Hadibarhaghtalab

“…Herein, we report the case of a 28-year-old woman with isolated vulvar LCH, misdiagnosed with herpes simplex infection, successfully treated with thalidomide.…”
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Medical Therapy of Steroid-Resistant Crohn's Disease by Bruce E Sands

“…Limited data are available on the use of tacrolimus, mycophenolate mofetil and thalidomide. Steroid-resistant Crohn’s disease, while involving a small minority of patients, continues to present a difficult therapeutic challenge.…”
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Microbiological Isolation of Sporothrix Schenckii in an Immunocompromised Patient. A Case Report by Ledys Pérez Morales, Midiala Iglesias López, Odette Quiñones Cherta, Idalmis Reyes Rodríguez

“…We present the case of 67-year-old white patient of rural origin with a history of myelofibrosis that had evolved for ten years and was treated with thalidomide and prednisone. He was admitted to the University General Hospital of Cienfuegos in August 2013 because of skin lesions on the left cheek, chest and forearms. …”
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Recurrent Obscure Gastrointestinal Bleeding: Dilemmas and Success with Pharmacological Therapies. Case Series and Review by Majid Almadi, Peter M Ghali, Andre Constantin, Jacques Galipeau, Andrew Szilagyi

“…In the third case, however, the addition of low-dose thalidomide stopped bleeding for a period of at least 23 months.…”
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Successful Use of Cyclophosphamide as an Add-On Therapy for Multiple Myeloma Patients with Acquired Resistance to Bortezomib or Lenalidomide by Shigeki Ito, Tatsuo Oyake, Kazunori Murai, Yoji Ishida

“…Novel agents such as thalidomide, lenalidomide, and bortezomib have been shown to possess potent activity against multiple myeloma. …”
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Plasmablastic Lymphoma: A Review of Current Knowledge and Future Directions by Ghaleb Elyamany, Eman Al Mussaed, Ali Matar Alzahrani

“…Recently, a few studies have reported the potential value of the proteasome inhibitor bortezomib and thalidomide in PBL patients. The introduction of genes encoding artificial receptors called chimeric antigen receptors (CARs) and CAR-modified T cells targeted to the B cell-specific CD19 antigen have demonstrated promising results in multiple early clinical trials. …”
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Acute Lymphoblastic Leukemia following Lenalidomide Maintenance for Multiple Myeloma: Two Cases with Unexpected Presentation and Good Prognostic Features by Abdullah M. Khan, Jameel Muzaffar, Hermant Murthy, John R. Wingard, Jan S. Moreb

“…Patients develop sALL after median of 32.5 months (range, 20–84) from being on lenalidomide or thalidomide maintenance, often presenting with cytopenias, display low tolerance to chemotherapy, but remission can often be achieved.…”
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Two Rare Complications in One Patient: Acquired von Willebrand Syndrome Associated with Intracranial Plasmacytoma by H. Auge, C. Yguel, E. Schmitt, B. Frotscher, H. Busby-Venner, R. Morizot, C. Moulin, P. Feugier, A. Perrot, L. Filliatre-Clement

“…The patient received induction therapy with bortezomib, thalidomide, and dexamethasone (VTD), followed by high-dose melphalan chemotherapy and autologous stem cell transplantation, and then VTD consolidation, and finally maintenance with lenalidomide. …”
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Synthesis, Characterization, and Biological Evaluation of Gabapentinoid Hybrids with Isoindole-1,3(2H)-Dione Moiety as Potential Antioxidant, Antimicrobial, and Anticancer Agents by Mirna Jabbour, Mohammad Ammar Al-Khayat, Mahmoud Al-Ktaifani

“…Compound 2 is highly effective against Caco-2 cells and more effective than thalidomide, with IC50 value less than 1 μmol/L. Conclusion. …”
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Use of Minimal Residual Disease Status to Reduce Uncertainty in Estimating Long-term Survival Outcomes for Newly Diagnosed Multiple Myeloma Patients by Naomi van Hest, Peter Morten, Keith Stubbs, Nicola Trevor

“…**Methods:** Standard and response-based PSMs, estimating patient life-years (LYs) over a lifetime horizon, were developed for NDMM patients treated with bortezomib, thalidomide, and dexamethasone (BTd) with or without daratumumab as induction and consolidation therapy. …”
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Histologic and Endoscopic Similarity between Nodular Gastric Antral Vascular Ectasia and Gastric Hyperplastic Polyps Potentially Causing Treatment Delays by Pujitha Kudaravalli, Sheikh A. Saleem, Rachana Mandru, Sekou Rawlins

“…There is not a lot of evidence supportive of pharmacological treatment of GAVE with estrogen-progesterone, tranexamic acid, and thalidomide. Serial endoscopic band ligation as well as detachable snares in the management of nodular GAVE refractory to argon plasma coagulation has also been tried. …”
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Lung-specific CRBN knockout attenuates influenza a virus-induced acute lung injury in mice: a potential therapeutic approach by Lifang Zhang, Qingchao Zhang, Jiahui Chang, Yunyi Zhou, Wei Wang, Xiliang Wang, Chengyu Jiang, Yanli Zhang

“…Cereblon (CRBN) is a protein known for its role in the ubiquitin-proteasome system and as a target of the drug thalidomide. However, the function of CRBN in influenza virus infection remains poorly understood. …”
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Clinical and biochemical characteristics for patients with polyneuropathy, organomegaly, endocrinopathy, M-protein, and skin changes syndrome: a pilot observational study by Pei Li, Ye Zhang, Li-Min Luo, Wen-Qing Wang, Jing Li, Yan Cheng, Xiao Dang, Yang Chen, Wei Jiang

“…VEGF level, which was negatively correlated with Ca2+ level (r = −0.56), was reduced in most patients with POEMS syndrome receiving bortezomib/ixazomib and lenalidomide/thalidomide therapy. Aspartate aminotransferase, total protein, and estimated glomerular filtration rate levels were increased, while creatinine and uric acid levels were reduced post-therapy in patients with POEMS syndrome.ConclusionPatients with POEMS syndrome had impaired liver and renal function, and effective therapy might partly repair the liver and renal dysfunction.…”
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Identification of MORF4L1 as an endogenous substrate of CRBN and its potential role as a therapeutic target in cancer by Luyao Zhang, Shunfang Liu, Jingliang He, Zhongke Hu, Lan Zhu, Hongyi Huang, Qi Gao, Dan Wang, Lu Chen, Xingyu Zhang, Ruotong Liu, Jiayun Wang, Yizhuo Song, Kaile Zeng, Xiuming Li, Yulu Chen, Xun Zou, Shaojie Ma, Xiujun Wang, Guofeng Xu, Wei Liu, Bin Liu

“…The cereblon (CRBN) E3 ubiquitin ligase complex is a crucial component of the UPS, particularly in modulating protein degradation in response to small-molecule modulators like thalidomide. However, the endogenous substrates of CRBN in solid tumors like HCC remain poorly characterized. …”
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