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https://www.readbyqxmd.com/read/29330393/haploidentical-bone-marrow-transplantation-with-post-transplant-cyclophosphamide-for-patients-with-x-linked-adrenoleukodystrophy-a-suitable-choice-in-an-urgent-situation
#1
Juliana Folloni Fernandes, Carmem Bonfim, Fábio Rodrigues Kerbauy, Morgani Rodrigues, Iracema Esteves, Nathalia Halley Silva, Alessandra Prandini Azambuja, Luiz Fernando Mantovani, José Mauro Kutner, Gisele Loth, Cilmara Cristina Kuwahara, Clarissa Bueno, Andrea Tiemi Kondo, Andreza Alice Feitosa Ribeiro, Fernando Kok, Nelson Hamerschlak
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment that enhances survival and stabilizes neurologic symptoms in X-linked adrenoleukodystrophy (X-ALD) with cerebral involvement, a severe demyelinating disease of childhood. Patients with X-ALD who lack a well-matched HLA donor need a rapid alternative. Haploidentical HSCT using post transplant cyclophosphamide (PT/Cy) has been performed in patients with malignant and nonmalignant diseases showing similar outcomes compared to other alternative sources...
January 12, 2018: Bone Marrow Transplantation
https://www.readbyqxmd.com/read/29329497/pharmacokinetic-interactions-between-elbasvir-grazoprevir-and-immunosuppressant-drugs-in-healthy-volunteers
#2
Hwa-Ping Feng, Luzelena Caro, Christine M Fandozzi, Zifang Guo, Jennifer Talaty, Dennis Wolford, Deborah Panebianco, Marian Iwamoto, Joan R Butterton, Wendy W Yeh
Elbasvir (EBR)/grazoprevir (GZR) may be coadministered with immunosuppressant drugs in posttransplant people who are infected with hepatitis C virus. The aim of the present study was to assess the safety and pharmacokinetic interactions between EBR and GZR and single doses of cyclosporine, tacrolimus, mycophenolate mofetil (MMF), and prednisone. This was a 4-part, open-label study in 58 healthy volunteers. Participants received single doses of cyclosporine 400 mg, tacrolimus 2 mg, MMF 1 g, or prednisone 40 mg alone or in the presence of once-daily EBR 50 mg/GZR 200 mg...
January 12, 2018: Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/29329489/mycophenolic-acid-and-its-metabolites-in-kidney-transplant-recipients-a-semimechanistic-enterohepatic-circulation-model-to-improve-estimating-exposure
#3
Malek Okour, Pamala A Jacobson, Mariam A Ahmed, Ajay K Israni, Richard C Brundage
Mycophenolic acid (MPA) is an approved immunosuppressive agent widely prescribed to prevent rejection after kidney transplantation. Wide between-subject variability (BSV) in MPA exposure exists which in part may be due to variability in enterohepatic recirculation (EHC). Several modeling strategies were developed to evaluate EHC as part of MPA pharmacokinetics, however mechanistic representation of EHC is limited. These models have not provided a satisfactory representation of the physiology of EHC in their modeling assumptions...
January 12, 2018: Journal of Clinical Pharmacology
https://www.readbyqxmd.com/read/29326307/mycophenolate-mofetil-in-combination-with-steroids-for-treatment-of-c3-glomerulopathy-a-case-series
#4
Rupali S Avasare, Pietro A Canetta, Andrew S Bomback, Maddalena Marasa, Yasar Caliskan, Yasemin Ozluk, Yifu Li, Ali G Gharavi, Gerald B Appel
BACKGROUND AND OBJECTIVES: C3 glomerulopathy is a form of complement-mediated GN. Immunosuppressive therapy may be beneficial in the treatment of C3 glomerulopathy. Mycophenolate mofetil is an attractive treatment option given its role in the treatment of other complement-mediated diseases and the results of the Spanish Group for the Study of Glomerular Diseases C3 Study. Here, we study the outcomes of patients with C3 glomerulopathy treated with steroids and mycophenolate mofetil. DESIGN, SETTING, PARTICIPANTS, & MEASUREMENTS: We conducted a retrospective chart review of patients in the C3 glomerulopathy registry at Columbia University and identified patients treated with mycophenolate mofetil for at least 3 months and follow-up for at least 1 year...
January 11, 2018: Clinical Journal of the American Society of Nephrology: CJASN
https://www.readbyqxmd.com/read/29325970/the-role-of-adjuvant-immunomodulatory-agents-for-treatment-of-severe-influenza
#5
REVIEW
David S Hui, Nelson Lee, Paul K Chan, John H Beigel
A severe inflammatory immune response with hypercytokinemia occurs in patients hospitalized with severe influenza, such as avian influenza A(H5N1), A(H7N9), and seasonal A(H1N1)pdm09 virus infections. The role of immunomodulatory therapy is unclear as there have been limited published data based on randomized controlled trials (RCTs). Passive immunotherapy such as convalescent plasma and hyperimmune globulin have some studies demonstrating benefit when administered as an adjunctive therapy for severe influenza...
January 8, 2018: Antiviral Research
https://www.readbyqxmd.com/read/29325675/mycophenolate-mofetil-as-a-therapeutic-agent-for-interstitial-lung-diseases-in-systemic-sclerosis
#6
REVIEW
Takahiro Ueda, Takuro Sakagami, Toshiaki Kikuchi, Toshinori Takada
Systemic sclerosis (SSc) is an intractable disease that causes fibrosis in all organs. Approximately 40% of patients with SSc have some degree of interstitial lung disease (ILD). One third of patients with SSc and ILD, approximately 15% of all patients, have pulmonary lesions, which slowly progress to respiratory failure resistant to corticosteroid and other treatments. A randomized controlled trial conducted in the United States indicated that one year of treatment with oral cyclophosphamide in patients with SSc-ILD had a significant but modest beneficial effect on lung function, dyspnea, thickening of the skin, and health-related quality of life...
January 2018: Respiratory Investigation
https://www.readbyqxmd.com/read/29321925/lupus-pneumonitis-presenting-with-high-titre-of-anti-ro-antibody
#7
Mei-Chuan Chen, Yueh-Lin Wu, Kai-Ling Lee, Kevin S Lai, Chi-Li Chung
Lupus pneumonitis carries high mortality and is a rare manifestation of systemic lupus erythematosus (SLE). However, it is difficult to diagnose and is often mistaken as pneumonia, alveolar haemorrhage, or organizing pneumonia. Previous studies demonstrated that serum anti-Ro antibodies are elevated more frequently in SLE patients with pneumonitis than in those without. We report a 21-year-old female who was newly diagnosed as having SLE with nephritis and who suddenly developed right lung opacity and rapidly progressed to severe hypoxaemia despite the use of broad-spectrum antibiotics...
January 2018: Respirology Case Reports
https://www.readbyqxmd.com/read/29320841/the-role-of-klotho-in-systemic-sclerosis
#8
R Talotta, S Bongiovanni, T Letizia, F Rigamonti, F Atzeni, M Benucci, T Vago, P Sarzi-Puttini
The aim was to evaluate the role of klotho in the pathogenesis of systemic sclerosis (SSc), through the measurement of its serum concentration in SSc patients compared to healthy controls, and to assess the association with cutaneous and visceral involvement. Blood samples obtained from both SSc patients and healthy controls were analysed by an ELISA assay for the detection of human klotho. SSc patients were globally evaluated for disease activity and assessed through the modified Rodnan's Skin Score, Medsger's scale, pulmonary function tests, 2D-echocardiography, nailfold capillaroscopy and laboratory tests...
December 21, 2017: Reumatismo
https://www.readbyqxmd.com/read/29319768/the-current-burden-of-cytomegalovirus-infection-in-kidney-transplant-recipients-receiving-no-pharmacological-prophylaxis
#9
Claudia Rosso Felipe, Alexandra Nicolau Ferreira, Adrieli Bessa, Tamiris Abait, Priscilla Ruppel, Mayara Ivani de Paula, Liliane Hiramoto, Laila Viana, Suelen Martins, Marina Cristelli, Wilson Aguiar, Juliana Mansur, Geovana Basso, Helio Tedesco Silva Junior, Jose Medina Pestana
Cytomegalovirus (CMV) infection in kidney transplantation has changed its clinical spectrum, mostly due to the current and more effective immunosuppression. In the absence of preventive strategies it is associated with significant morbi-mortality. OBJECTIVE: This study evaluated the incidence of CMV events and its effect on outcomes of kidney transplantation in recipients without pharmacological prophylaxis or targeted preemptive treatment. RESULTS: The study cohort comprised 802 recipients of kidney transplants between 04/30/2014 and 04/30/2015...
October 2017: Jornal Brasileiro de Nefrologia: ʹorgão Oficial de Sociedades Brasileira e Latino-Americana de Nefrologia
https://www.readbyqxmd.com/read/29314776/chronicity-index-especially-glomerular-sclerosis-is-the-most-powerful-predictor-of-renal-response-following-immunosuppressive-treatment-in-patients-with-lupus-nephritis
#10
Dong-Jin Park, Sung-Eun Choi, Haimuzi Xu, Ji-Hyoun Kang, Kyung-Eun Lee, Ji Shin Lee, Yoo-Duk Choi, Shin-Seok Lee
AIM: Renal responses to immunosuppressive agents in patients with lupus nephritis (LN) differ depending on ethnicity, follow-up duration, disease severity and treatment. Thus, we evaluated predictors of complete remission during the first year following immunosuppressive treatment in patients with LN. METHODS: We retrospectively reviewed 79 patients who underwent kidney biopsy prior to the start of induction treatment and who were subsequently treated with immunosuppressive drugs for at least 6 months and followed-up for more than a year...
January 5, 2018: International Journal of Rheumatic Diseases
https://www.readbyqxmd.com/read/29308669/an-open-label-study-of-a-novel-immunosuppression-intervention-for-the-treatment-of-amyotrophic-lateral-sclerosis
#11
Christina N Fournier, David Schoenfeld, James D Berry, Merit E Cudkowicz, James Chan, Colin Quinn, Robert H Brown, Johnny S Salameh, Malu G Tansey, David R Beers, Stanley H Appel, Jonathan D Glass
Neuroinflammation is increasingly tied to disease progression in amyotrophic lateral sclerosis (ALS). Participants in the first-in-human trial of intra-spinal allogeneic stem cell therapy for ALS received immunosuppression, and one participant saw dramatic improvement across multiple outcome measures. The primary objective of this study (NCT01884571) was to assess the rate of clinical response to the same immunosuppressive regimen using basiliximab, tacrolimus, mycophenolate, and prednisone in people with ALS...
January 8, 2018: Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration
https://www.readbyqxmd.com/read/29308550/mycophenolic-acid-induces-differentiation-of-toxoplasma-gondii-rh-strain-tachyzoites-into-bradyzoites-and-formation-of-cyst-like-structure-in-vitro
#12
Kitzia N Castro-Elizalde, Pedro Hernández-Contreras, Carlos J Ramírez-Flores, Sirenia González-Pozos, Carmen T Gómez de León, Mónica Mondragón-Castelán, Ricardo Mondragón-Flores
The biochemical and structural changes that occur during the conversion of Toxoplasma gondii tachyzoites to bradyzoites and the formation of tissue cyst are not well understood. Maintaining cells infected with T. gondii type II and III strains under stress conditions induces the tachyzoite-bradyzoite in vitro differentiation, along with the formation of cyst-like structures. However, due to the long exposure to such conditions required to induce the differentiation, the severe damages in the host cell and the low encystation frequency, it has been difficult to dissect in more detail these processes...
January 8, 2018: Parasitology Research
https://www.readbyqxmd.com/read/29308284/an-unusual-flare-of-anti-synthetase-syndrome-during-concurrent-trastuzumab-therapy-given-for-recurrent-breast-cancer
#13
Timothy D Reynolds, Vivek Mohan, Matthew Roy, Nathan Manghat, Huzaifa Adamali, Harsha Gunawardena
We present the case of a patient with relapsing anti-synthetase syndrome (ASS) that may have been triggered by monoclonal antibody trastuzumab therapy given for breast cancer. A 52-year-old female with a history of anti-Jo1-associated ASS went into remission with glucocorticoids and mycophenolate mofetil. Her past history included invasive ductal carcinoma of the right breast that was fully treated six years prior to the onset of ASS. She subsequently developed recurrent right-sided breast cancer that was treated with right mastectomy and six cycles of cyclophosphamide-docetaxel chemotherapy...
December 2017: European Journal of Rheumatology
https://www.readbyqxmd.com/read/29307718/haploidentical-stem-cell-transplantation-for-refractory-relapsed-neuroblastoma
#14
Toni Illhardt, Jacek Toporski, Tobias Feuchtinger, Dominik Turkiewicz, Heiko-Manuel Teltschik, Martin Ebinger, Carl-Philipp Schwarze, Ursula Holzer, Holger N Lode, Michael H Albert, Bernd Gruhn, Christian Urban, Josefina H Dykes, Oliver Teuffel, Michael Schumm, Rupert Handgretinger, Peter Lang
BACKGROUND: Pediatric patients with refractory or relapsed metastatic neuroblastoma (NBL) have a poor prognosis despite autologous stem cell transplantation (SCT). Allogeneic SCT from haploidentical donors had remarkable alloreactive effects in leukemia patients; hence, we evaluated this approach in children with very high-risk NBL. METHODS: We analyzed data from two prospective phase I/II trials. 26 patients with refractory (n=5) or metastatic relapsed (n=20) or locally relapsed MYCN+ (n=1) neuroblastoma received a median of 17x106/kg T/B cell depleted CD34+ stem cells with 68x103/kg residual T cells and 107x106/kg NK cells...
January 4, 2018: Biology of Blood and Marrow Transplantation
https://www.readbyqxmd.com/read/29306856/mycophenolate-mofetil-treatment-in-a-patient-with-recurrent-lymphocytic-hypophysitis
#15
Hiroshi Sawachika, Shoko Kodama, Tomoyuki Mukai, Yoshitaka Morita
Lymphocytic hypophysitis (LHP) is a relatively rare disease characterised by lymphocytic infiltration of the pituitary gland, resulting in pituitary dysfunction. LHP is generally responsive to corticosteroid therapy, but cases with recurrence require clinicians to select second-line therapy. We report here the case of a 58-year-old patient with LHP who developed panhypopituitarism and bitemporal hemianopia. He responded to prednisolone 40 mg/day but relapsed during tapering. The prednisolone dose was increased again and mycophenolate mofetil (MMF) was added...
January 6, 2018: BMJ Case Reports
https://www.readbyqxmd.com/read/29305608/disease-course-and-treatment-responses-in-children-with-relapsing-myelin-oligodendrocyte-glycoprotein-antibody-associated-disease
#16
Yael Hacohen, Yu Yi Wong, Christian Lechner, Maciej Jurynczyk, Sukhvir Wright, Bahadir Konuskan, Judith Kalser, Anne Lise Poulat, Helene Maurey, Esther Ganelin-Cohen, Evangeline Wassmer, Chery Hemingway, Rob Forsyth, Eva Maria Hennes, M Isabel Leite, Olga Ciccarelli, Banu Anlar, Rogier Hintzen, Romain Marignier, Jacqueline Palace, Matthias Baumann, Kevin Rostásy, Rinze Neuteboom, Kumaran Deiva, Ming Lim
Importance: Myelin oligodendrocyte glycoprotein antibodies (MOG-Abs) are consistently identified in a range of demyelinating disorders in adults and children. Current therapeutic strategies are largely center specific, and no treatments have been formally evaluated. Objective: To examine the clinical phenotypes, treatment responses, and outcomes of children with relapsing MOG-Ab-associated disease. Design, Setting, and Participants: This study prospectively collected demographic, clinical, and radiologic data from 102 patients from 8 countries of the EU Paediatric Demyelinating Disease Consortium from January 1, 2014, through December 31, 2016...
January 5, 2018: JAMA Neurology
https://www.readbyqxmd.com/read/29303612/the-effect-of-induction-therapy-on-established-cmv-specific-t-cell-immunity-in-living-donor-kidney-transplantation
#17
L Stranavova, P Hruba, E Girmanova, I Tycova, A Slavcev, J Fronek, J Slatinska, P Reinke, H-D Volk, O Viklicky
Cytomegalovirus (CMV) infection influences both short and long term outcomes in immunosuppressed organ transplant recipients. The aim of this study was to evaluate the effect of different induction immunosuppression regimens on CMV specific T cell response in patients with already established CMV immunity. In 24 seropositive living donor kidney recipients, the frequency of CMV specific T cells was determined by ELISPOT (Enzyme-Linked ImmunoSpot) assay prior and 6 months after transplantation. Recipients' peripheral blood mononuclear cells were stimulated with immediate-early (IE1) and phosphoprotein 65 (pp65) CMV-derived peptide pools and the number of cells producing interferon gamma (IFN-gamma) was assessed...
January 5, 2018: Physiological Research
https://www.readbyqxmd.com/read/29301801/clinically-amyopathic-dermatomyositis-associated-with-anti-mda5-antibody
#18
Konstantinos Parperis, Amirali Kiyani
Clinically amyopathic dermatomyositis (CADM) is a rare entity that presents with cutaneous manifestations of classic dermatomyositis but without muscle weakness or abnormal muscle enzymes. It is more common in young white and Asian females. A subset of patients with CADM has a specific antibody known as anti-MDA5. These patients have a more aggressive course with distinct cutaneous features, pulmonary involvement and early death. Here, we present the case of a 64-year-old Caucasian male with no significant medical history who was admitted with marked weight loss and a painful rash for 6 months...
January 4, 2018: BMJ Case Reports
https://www.readbyqxmd.com/read/29297205/cyclophosphamide-for-connective-tissue-disease-associated-interstitial-lung-disease
#19
REVIEW
Hayley Barnes, Anne E Holland, Glen P Westall, Nicole Sl Goh, Ian N Glaspole
BACKGROUND: Approximately one-third of individuals with interstitial lung disease (ILD) have associated connective tissue disease (CTD). The connective tissue disorders most commonly associated with ILD include scleroderma/systemic sclerosis (SSc), rheumatoid arthritis, polymyositis/dermatomyositis, and Sjögren's syndrome. Although many people with CTD-ILD do not develop progressive lung disease, a significant proportion do progress, leading to reduced physical function, decreased quality of life, and death...
January 3, 2018: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/29289665/disulfiram-can-inhibit-mers-and-sars-coronavirus-papain-like-proteases-via-different-modes
#20
Min-Han Lin, David C Moses, Chih-Hua Hsieh, Shu-Chun Cheng, Yau-Hung Chen, Chiao-Yin Sun, Chi-Yuan Chou
Severe acute respiratory syndrome coronavirus (SARS-CoV) emerged in southern China in late 2002 and caused a global outbreak with a fatality rate around 10% in 2003. Ten years later, a second highly pathogenic human CoV, MERS-CoV, emerged in the Middle East and has spread to other countries in Europe, North Africa, North America and Asia. As of November 2017, MERS-CoV had infected at least 2102 people with a fatality rate of about 35% globally, and hence there is an urgent need to identify antiviral drugs that are active against MERS-CoV...
December 28, 2017: Antiviral Research
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