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https://www.readbyqxmd.com/read/27913466/nontransplant-therapy-for-bone-marrow-failure
#1
Danielle M Townsley, Thomas Winkler
Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27905115/treatment-for-hereditary-angioedema-with-normal-c1-inh-and-specific-mutations-in-the-f12-gene-hae-fxii
#2
K Bork, K Wulff, G Witzke, J Hardt
Hereditary angioedema with normal C1 esterase inhibitor and mutations in the F12 gene (HAE-FXII) is associated with skin swellings, abdominal pain attacks, and the risk of asphyxiation due to upper airway obstruction. It occurs nearly exclusively in women. We report our experience treating HAE-FXII with discontinuation of potential trigger factors and drug therapies. The study included 72 patients with HAE-FXII. Potential triggers included estrogen-containing oral contraceptives (eOC), hormonal replacement therapy, or angiotensin-converting enzyme inhibitors...
November 6, 2016: Allergy
https://www.readbyqxmd.com/read/27864873/hereditary-haemorrhagic-telangiectasia-to-transplant-or-not-to-transplant-is-there-a-right-time-for-liver-transplantation
#3
Yannick D Muller, Roland Oppliger, Romain Breguet, Philippe Meyer, Laura Rubbia-Brandt, Pierre-Auguste Petignat, Thomas Harr, Eric Dayer, Jörg D Seebach
BACKGROUND & AIMS: Hereditary haemorrhagic telangiectasia is characterized by arterio-venous malformations (AVM). It frequently involves the liver without clinical symptoms, but may lead to biliary ischaemia, portal hypertension, or fatal high-output heart failure. The indication of liver transplantation is controversial. METHODS: Herein, we report the case of a 65-year-old female patient with a 'double Osler syndrome' consisting of hereditary haemorrhagic telangiectasia (HHT) and type I hereditary angioedema diagnosed at the age of 25 and 22 years respectively...
December 2016: Liver International: Official Journal of the International Association for the Study of the Liver
https://www.readbyqxmd.com/read/27774820/myelofibrosis-an-update-on-drug-therapy-in-2016
#4
Prithviraj Bose, Srdan Verstovsek
Primary myelofibrosis (PMF) is the least common but the most aggressive of the classic Philadelphia chromosome-negative myeloproliferative neoplasms. Survival is much shorter in PMF than in polycythemia vera (PV) or essential thrombocythemia (ET). Post-PV/ET myelofibrosis (MF) is clinically indistinguishable from PMF and approached similarly. Areas covered: Current pharmacologic therapy of MF revolves around the Janus kinase 1/2 (JAK1/2) inhibitor ruxolitinib, which dramatically improves constitutional symptoms and splenomegaly in the majority of patients, and improves overall survival (OS)...
November 7, 2016: Expert Opinion on Pharmacotherapy
https://www.readbyqxmd.com/read/27626530/danazol-treatment-for-telomere-diseases
#5
LETTER
Stéphane Jouneau, Mallorie Kerjouan, Charles Ricordel
No abstract text is available yet for this article.
September 15, 2016: New England Journal of Medicine
https://www.readbyqxmd.com/read/27626529/danazol-treatment-for-telomere-diseases
#6
LETTER
Mathis Grossmann
No abstract text is available yet for this article.
September 15, 2016: New England Journal of Medicine
https://www.readbyqxmd.com/read/27626528/danazol-treatment-for-telomere-diseases
#7
LETTER
Danielle M Townsley, Bogdan Dumitriu, Neal S Young
No abstract text is available yet for this article.
September 15, 2016: New England Journal of Medicine
https://www.readbyqxmd.com/read/27607446/allogeneic-hematopoietic-stem-cell-transplantation-for-dyskeratosis-congenita
#8
Shaimaa Elmahadi, Hideki Muramatsu, Seiji Kojima
PURPOSE OF REVIEW: Dyskeratosis congenita is an inherited bone marrow failure syndrome caused by defects in telomere maintenance. Hematopoietic stem cell transplantation (HSCT) is the only curative treatment for bone marrow failure because of dyskeratosis congenita. The present review summarizes the literature with respect to the diagnosis and treatment of patients with dyskeratosis congenita who received HSCT, and discusses the recent progress in the management of dyskeratosis congenita...
November 2016: Current Opinion in Hematology
https://www.readbyqxmd.com/read/27599419/danazol-induced-peliosis-hepatis-accompanied-by-disseminated-intravascular-coagulation-in-a-patient-with-myelodysplastic-syndrome-transformed-from-aplastic-anemia
#9
Yasuhiro Tsukamoto, Junichi Kiyasu, Hayato Utsunomiya, Yasuhiro Nakashima, Ilseung Choi, Youko Suehiro, Yoshifusa Aratake, Yasunobu Abe
Peliosis hepatis (PH) is a condition involving benign tumors pathologically characterized by multiple blood-filled cavities, mostly affecting the liver and spleen. Androgenic-steroids are widely used in patients with bone marrow failure syndromes (e.g.: aplastic anemia) and these patients are at increased risk of developing PH. Although patients with PH are generally asymptomatic, PH can progress to liver failure and even fatal spontaneous intraabdominal hemorrhage. Therefore, early diagnosis is critical in order to prevent life-threatening complications of PH...
August 2016: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/27533712/in-vivo-precipitation-of-poorly-soluble-drugs-from-lipid-based-drug-delivery-systems
#10
P J Sassene, M H Michaelsen, M D Mosgaard, M K Jensen, E Van Den Broek, K M Wasan, H Mu, T Rades, A Müllertz
Precipitation of poorly water-soluble drugs from lipid-based drug delivery systems (LbDDS) has been studied extensively during in vitro lipolysis but has never been shown in vivo. The aim of this study was therefore to investigate if drug precipitation can occur from LbDDS during transit of the gastrointestinal tract in vivo. Rats were administered 300 μL of either of two LbDDS (LbDDS I and LbDDS II) loaded with danazol or fenofibrate (or paracetamol to assess gastric emptying). The rats were euthanized at various time points after administration of both LbDDS containing either drug, and the contents of the stomach and proximal part of the small intestine were harvested...
October 3, 2016: Molecular Pharmaceutics
https://www.readbyqxmd.com/read/27503693/hormonal-therapy-for-endometriosis-from-molecular-research-to-bedside
#11
Claudia Tosti, Antonella Biscione, Giuseppe Morgante, Giuseppe Bifulco, Stefano Luisi, Felice Petraglia
Endometriotic lesions are associated with hormonal imbalance, including increased estrogen synthesis, metabolism and progesterone resistance. These hormonal changes cause increased proliferation, inflammation, pain and infertility. Hormonal imbalances are targets for treatment. Therapeutic strategies and innovations of hormonal drugs for endometriosis are increasing. Acting on estrogen receptors are hormonal drugs decreasing systemic and local estrogen synthesis (GnRH analogs, GnRH antagonists, Aromatase inhibitors) or estrogen activity (selective estrogen receptor modulators)...
May 27, 2016: European Journal of Obstetrics, Gynecology, and Reproductive Biology
https://www.readbyqxmd.com/read/27488902/studying-the-propensity-of-compounds-to-supersaturate-a-practical-and-broadly-applicable-approach
#12
Henrik Palmelund, Cecilie Maria Madsen, Jakob Plum, Anette Müllertz, Thomas Rades
Supersaturating drug delivery systems can enhance the oral bioavailability of poorly soluble drug compounds. Supersaturation of such compounds has been studied in many different ways; however, a more standardized method is required. The rationale of choosing suitable concentrations of supersaturation to study has previously been very inconsistent. This makes comparisons between studies and compounds difficult, as the propensity of compounds to supersaturate varies greatly. This study presents a standardized method to study the supersaturation of drug compounds...
October 2016: Journal of Pharmaceutical Sciences
https://www.readbyqxmd.com/read/27394004/analysis-of-sulfate-metabolites-of-the-doping-agents-oxandrolone-and-danazol-using-high-performance-liquid-chromatography-coupled-to-tandem-mass-spectrometry
#13
S Rzeppa, L Viet
The direct detection of sulfate conjugates of anabolic androgenic steroids (AAS) can be a powerful tool in doping control analysis. By skipping the solvolysis step analysis time can be reduced, and due to long term sulfate metabolites the detection time can be significantly extended as demonstrated for some AAS. This study presents the successful identification of sulfate metabolites of the doping agents oxandrolone and danazol in excretion urines by high performance liquid chromatography coupled to tandem mass spectrometry (HPLC-MS/MS)...
September 1, 2016: Journal of Chromatography. B, Analytical Technologies in the Biomedical and Life Sciences
https://www.readbyqxmd.com/read/27379972/adenomyosis-a-systematic-review-of-medical-treatment
#14
A Pontis, M N D'Alterio, S Pirarba, C de Angelis, R Tinelli, S Angioni
Adenomyosis is a heterogeneous gynaecologic condition with a range of clinical presentations, the most common being heavy menstrual bleeding and dysmenorrhoea; however, patients can also be asymptomatic. Several studies support the theory that adenomyosis results from invasion of the endometrium into the myometrium, causing alterations in the junctional zone. These changes are commonly seen on imaging studies, such as transvaginal ultrasound and magnetic resonance imaging. The aim of this review is to discuss the medical approach to the management of adenomyosis symptoms, including pain and abnormal uterine bleeding...
July 5, 2016: Gynecological Endocrinology
https://www.readbyqxmd.com/read/27364943/thyroid-hormones-and-complement-parameters-in-hereditary-angioedema-with-c1-inhibitor-deficiency
#15
Ibolya Czaller, Dorottya Csuka, Zsuzsanna Zotter, Nóra Veszeli, Edit Takács, Éva Imreh, Lilian Varga, Henriette Farkas
BACKGROUND: Thyroid hormones control and up-regulate the synthesis of many plasma proteins. OBJECTIVE: To explore possible associations between thyroid hormone and complement levels in patients with hereditary angioedema resulting from the deficiency of the C1-inhibitor (C1-INH-HAE). METHODS: In this case-control study, serum thyrotropin, free triiodothyronine (FT3), and free thyroxine (FT4) levels, anti-thyroid peroxidase and antithyroglobulin antibody titers, and C1-INH concentrations were measured in 117 euthyroid patients with C1-INH-HAE and compared with their clinical properties...
August 2016: Annals of Allergy, Asthma & Immunology
https://www.readbyqxmd.com/read/27320967/development-of-hepatocellular-carcinoma-after-long-term-immunosuppressive-therapy-including-danazol-in-a-dog
#16
Kosuke Kobayashi, Takako Shimokawa Miyama, Kazuhito Itamoto, Shunsuke Noguchi, Kenji Baba, Takuya Mizuno, Masaru Okuda
A 2-year-old female beagle was referred to our hospital for evaluation of anemia. Laboratory tests, including bone marrow cytology, revealed non-regenerative immune-mediated anemia (NRIMA). Although initial immunosuppressive multi-drug therapy was not effective, additional administration of danazol was successful in treating the anemia. However, hepatocellular carcinoma (HCC) developed about 20 months after the administration of danazol. In humans, several cases of development of HCC after the administration of danazol have been reported...
June 20, 2016: Journal of Veterinary Medical Science
https://www.readbyqxmd.com/read/27293138/extended-duration-strategies-for-the-pharmacologic-treatment-of-diabetic-retinopathy-current-status-and-future-prospects
#17
Michael W Stewart, Harry W Flynn, Stephen G Schwartz, Ingrid U Scott
INTRODUCTION: Intraocular pharmacotherapy (vascular endothelial growth factor [VEGF] inhibitors and corticosteroids) has become first-line therapy for diabetic retinopathy (DR). A series of intraocular injections is usually required before disease modulation decreases the treatment burden in some patients, but others with chronic diabetic macular edema may require intensive longer-term therapy. AREAS COVERED: Recent studies showing successful pharmacologic treatment of proliferative DR will probably lead to increased use of pharmacotherapy, thereby further emphasizing the need for longer duration drugs...
September 2016: Expert Opinion on Drug Delivery
https://www.readbyqxmd.com/read/27277147/treatment-in-idiopathic-thrombocytopenic-purpura-in-the-elderly-about-a-retrospective-study
#18
Abrar-Ahmad Zulfiqar, Jean-Luc Novella, Rachid Mahmoudi, Jean-Loup Pennaforte, Emmanuel Andres
There are no specific recommendations on the care of elderly patients with ITP. A retrospective study over two academic centers (Reims and Strasbourg), focused on patients over 65 who have been diagnosed idiopathic thrombocytopenic purpura in two unit of internal medicine. 41 patients were enrolled, including 27 women (66%). The median age is 76.75 years. On admission, the average thrombocytopenia is 34.45 G/L; 24 patients (58.5%) showed severe bleeding mucosal and/or visceral signs; 37 patients (90%) had a first-line treatment...
June 1, 2016: Gériatrie et Psychologie Neuropsychiatrie du Vieillissement
https://www.readbyqxmd.com/read/27225336/dienogest-compared-with-gonadotropin-releasing-hormone-agonist-after-conservative-surgery-for-endometriosis
#19
Yotaro Takaesu, Hirotaka Nishi, Junya Kojima, Toru Sasaki, Yuzo Nagamitsu, Rina Kato, Keiichi Isaka
AIM: Although there are various hormone therapies, including gonadotropin-releasing hormone agonist, danazol, levonorgestrel-releasing intrauterine system, dienogest, and low-dose estrogen progestin, no consensus opinion has been reached in terms of which medication should be used and for how long it should be administered. We aimed to determine whether dienogest or goserelin is the better postoperative therapy to prevent recurrence of endometriosis. METHODS: A prospective cohort randomized study were conducted, including 198 patients diagnosed as having endometriosis...
September 2016: Journal of Obstetrics and Gynaecology Research
https://www.readbyqxmd.com/read/27192671/danazol-treatment-for-telomere-diseases
#20
Danielle M Townsley, Bogdan Dumitriu, Delong Liu, Angélique Biancotto, Barbara Weinstein, Christina Chen, Nathan Hardy, Andrew D Mihalek, Shilpa Lingala, Yun Ju Kim, Jianhua Yao, Elizabeth Jones, Bernadette R Gochuico, Theo Heller, Colin O Wu, Rodrigo T Calado, Phillip Scheinberg, Neal S Young
BACKGROUND: Genetic defects in telomere maintenance and repair cause bone marrow failure, liver cirrhosis, and pulmonary fibrosis, and they increase susceptibility to cancer. Historically, androgens have been useful as treatment for marrow failure syndromes. In tissue culture and animal models, sex hormones regulate expression of the telomerase gene. METHODS: In a phase 1-2 prospective study involving patients with telomere diseases, we administered the synthetic sex hormone danazol orally at a dose of 800 mg per day for a total of 24 months...
May 19, 2016: New England Journal of Medicine
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