Purpose:This article summarizes data submitted to the U.S. Food and Drug Administration for marketing approval of azacitidine as injectable suspension (Vidaza, Pharmion Corporation, Boulder, CO) for treatment of patients with myelodysplastic syndrome. Experimental Design: In one phase 3 controlled trial, 191 study subjects were randomized to treatment with azacitidine or to observation; an additional 120 patients were treated with azacitidine in two phase 2 single arm studies. The primary efficacy end point was the overall response rate, defined as complete or partial normalization of peripheral blood counts and bone marrow blast percentages for at least 4 weeks. Results: In the controlled trial, the overall response rate was 15.7% in the azacitidine treatment group; there were no responders in the observation group (P < 0.0001). Response rates were similar in the two single arm studies. During response patients stopped being red cell or platelet transfusion dependent. Median duration of responses was at least 9 months. An additional 19% of azacitidine-treated patients had less than partial responses, most becoming transfusion independent. The most common adverse events attributed to azacitidine were gastrointestinal, hematologic, local (injection site), and constitutional. There were no azacitidine-related deaths. Conclusions: On May 19, 2004 the U.S. Food and Drug Administration approved azacitidine as injectable suspension for treatment of patients with the following myelodysplastic syndrome subtypes: refractory anemia or refractory anemia with ringed sideroblasts (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, and chronic myelomonocytic leukemia. Full prescribing information is available at http://www.fda.gov/cder/foi/label/2004/050794lbl.pdf. Azacitidine is the first agent approved for treatment of myelodysplastic syndrome.

Increased red blood cell (RBC) volume variation (RDW) has recently been shown to predict a wide range of mortality and morbidity: death due to cardiovascular disease, cancer, infection, renal disease, and more; complications in heart failure and coronary artery disease, advanced stage and worse prognosis in many cancers, poor outcomes in autoimmune disease, and many more. The mechanisms by which all of these diseases lead to increased RDW are unknown. Here we use a semi-mechanistic mathematical model of in vivo RBC population dynamics to dissect the factors controlling RDW and show that elevated RDW results largely from a slight reduction in the in vivo rate of RBC turnover. RBCs become smaller as they age, and a slight reduction in the rate of RBC turnover allows smaller cells to continue circulating, expanding the low-volume tail of the RBC population’s volume distribution, and thereby increasing RDW. Our results show that mildly extended RBC lifespan is a previously unrecognized homeostatic adaptation common to a very wide range of pathologic states, likely compensating for subtle reductions in erythropoietic output. A mathematical model-based estimate of the clearance rate may provide a novel early-warning biomarker for a wide range of morbidity and mortality.

Bifrontal decompressive craniectomy has been used on an ad hoc basis for the treatment of post-traumatic intracranial hypertension for more than thirty years. In this observational study we report the clinical outcome and physiological effects of the procedure in a series of 26 patients with refractory intracranial hypertension treated on a protocol driven basis. Bifrontal decompressive craniectomy was associated with significant reductions in mean ICP from 37.5 to 18.1 mmHg (p = 0.003). In addition, craniectomy reduced the amplitude of ICP waves (p < 0.02) and increased compensatory reserve (p < 0.05). A favourable outcome was achieved in 69% of patients; 8% were severely disabled and 23% died. We conclude that this study provides pathophysiological evidence that bifrontal decompressive craniectomy significantly reduces posttraumatic intracranial hypertension and improves pressure dynamics. Our results support the continued use of bifrontal decompressive craniectomy in selected patients after head injury.

A prospective study was performed to compare the efficacy and patient tolerance of Merocel and Rapid Rhino nasal tampons in the treatment of epistaxis. A total of 42 patients were studied. There was no significant difference between the two types of pack in efficacy or patient discomfort with pack in situ. Rapid Rhino produced significantly lower scores for subjective patient discomfort during insertion and removal of pack.

Introduction The 2001 Recommendations for clinical care guidelines on the management of otitis media in Aboriginal and Torres Islander populations were revised in 2010. This 2020 update by the Centre of Research Excellence in Ear and Hearing Health of Aboriginal and Torres Strait Islander Children used for the first time the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) approach. Main recommendations We performed systematic reviews of evidence across prevention, diagnosis, prognosis and management. We report ten algorithms to guide diagnosis and clinical management of all forms of otitis media. The guidelines include 14 prevention and 37 treatment strategies addressing 191 questions. Changes in management as a result of the guidelines A GRADE approach is used. Targeted recommendations for both high and low risk children. New tympanostomy tube otorrhoea section. New Priority 5 for health services: annual and catch‐up ear health checks for at‐risk children. Antibiotics are strongly recommended for persistent otitis media with effusion in high risk children. Azithromycin is strongly recommended for acute otitis media where adherence is difficult or there is no access to refrigeration. Concurrent audiology and surgical referrals are recommended where delays are likely. Surgical referral is recommended for chronic suppurative otitis media at the time of diagnosis. The use of autoinflation devices is recommended for some children with persistent otitis media with effusion. Definitions for mild (21–30 dB) and moderate (> 30 dB) hearing impairment have been updated. New “OMapp” enables free fast access to the guidelines, plus images, animations, and multiple Aboriginal and Torres Strait Islander language audio translations to aid communication with families.

We evaluated a new method of assessing patients referred to the otolaryngology department with nasal injury. Patients possessing a mobile phone with built-in camera took an image of their face and sent it to the department for assessment. A decision was then made as to whether the patient needed further consultation or treatment. This method of nasal fracture assessment might avoid unnecessary referrals to the department. A prospective single blinded study was carried out. Twenty-five patients with a nasal injury took photographs of their face using a mobile phone camera. These images were reviewed and assessment made about whether a nasal fracture was present. The patient was then clinically assessed, the clinical examination being the 'gold standard' method of assessment. There was little agreement between photographic and clinical assessment. Sixty-two percent of patients who were clinically assessed to have a nasal fracture requiring manipulation were not picked up on assessment of their image. The greatest agreement with clinical assessment was the patient's own opinion as to whether there was new deviation of their nasal bone.

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Introduction: Surgical ligation is used in the treatment of painful varicocele. We conducted this retrospective study to establish the effectiveness of varicocele ligation for the treatment of pain and to examine the factors that might predict outcomes. Patients and Methods: Eighty-three patients underwent varicocele ligation for pain during the study period. Review of medical records and postal questionnaires were used to document patient age, grade and location of varicocele, duration and quality of pain, surgical approach, complications and the resolution of pain. Results: Follow-up was available for 68 (82%) patients. In 76.5% patients there was marked or complete resolution of pain and 14 had partial resolution. Only 9% patients had persistent pain and 1.5% patient reported worsening pain. The quality of pain, dull ache or dragging, was the only factor that correlated with the resolution of pain following surgery. Conclusions: Surgical ligation is an effective treatment for the painful varicocele. The quality of pain at presentation may predict outcomes in selected patients.