Cost of illness for neuromuscular diseases in the United States

Introduction: We conducted a comprehensive study of the costs associated with amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy (DMD). and myotonic dystrophy (DM) in the U.S. Methods: We determined the total impact on the U.S. economy, including direct medical costs, nonmedical costs, and loss of income. Medical costs were calculated using a commercial insurance database and Medicare claims data. Nonmedical and indirect costs were determined through a survey of families registered with the Muscular Dystrophy Association. Results: Medical costs were driven by outpatient care. Nonmedical costs were driven by the necessity to move or adapt housing for the patient and paid caregiving. Loss of income correlated significantly with the amount of care needed by the patient. Conclusions: We calculated the annual per‐patient costs to be $63,693 for ALS, $50,952 for DMD, and $32,236 for DM. Population‐wide national costs were $1,023 million (ALS), $787 million (DMD), and $448 million (DM). Muscle Nerve 49 :431–438, 2014     

Prevalence of hepatitis B and hepatitis C viral infections in Indian patients with chronic renal failure

The present study reports prevalence of posttransplant hepatitis B virus (HBV) and hepatitis C virus (HCV) infection in 256 patients with chronic renal failure (CRF) and with a history of either renal transplant or hemodialysis. Out of 256 patients 138 had renal transplant and 118 were on maintenance hemodialysis. Among the patients screened, 7% had HBV infection alone, 46% were infected with HCV alone, while 37.10% were found to have co-infection of both the viruses. Our findings implicate these viruses as the major cause of posttransplant hepatitis in Indian patients with CRF and indicate implementation of stringent screening procedures for these two viral infections.     

Characteristics of white cell-reduced red cells stored in tri-(2- ethylhexyl)trimellitate plastic

BACKGROUND: Standard blood storage containers contain extractable plasticizers that accumulate in blood during storage and are an unintended transfusion product. However, extractable plasticizers have a protective effect on the red cell membrane and improve red cell storage variables. Prestorage white cell reduction also improves selected red cell storage variables. STUDY DESIGN AND METHODS: The study evaluated whether the beneficial effect of prestorage white cell reduction would offset the negative effect of the absence of extractable plasticizer in red cells stored in AS-3 for 42 days at 4 degrees C. Filtered red cells stored in polyvinylchloride containers with the nonextracting plasticizer, tri-(2-ethylhexyl)trimellitate (TEHTM), were compared to unfiltered red cells stored in polyvinylchloride containers with the extractable plasticizer di-(2- ethylhexyl)phthalate (DEHP). RESULTS: Poststorage supernatant potassium and red cell osmotic fragility were significantly higher in white cell- reduced TEHTM units than in unfiltered DEHP units. The mean 24-hour recovery of the filtered TEHTM red cells was significantly lower than that of the unfiltered DEHP red cells (69.1 +/− 7.4% vs. 77.1 +/− 5.1%, p < 0.05, n = 8). CONCLUSION: These data demonstrate that white cell reduction before 42-day storage in TEHTM containers with currently approved preservatives does not yield an acceptable red cell component.     

DNA ligation in relation to DNA strand breaks in phytohemagglutinin- stimulated blast cells from acute lymphoblastic and nonlymphoblastic leukemia

DNA ligase activity was determined in the WBCs from 306 cases of acute lymphoblastic leukemia (ALL) and acute nonlymphocytic leukemia (ANLL). In T-ALL cells this activity was either low or absent. DNA analysis by nucleoid, alkaline elution, and alkaline sucrose centrifugation after cells were embedded in agarose inserts has shown more DNA breaks in T- ALL than in ANLL blasts. Phytohemagglutinin stimulation of T-ALL blasts resulted in the apparent joining of the DNA breaks. Apparent identical results can be obtained by the incubation of DNA with exogenous DNA ligase. The authors suggest that this enzyme is a crucially regulated step of replication and subsequent proliferation in this type of leukemia.     

Does growth hormone treatment of patients with Turner's syndrome cause an abnormal body shape?

The effect of human growth hormone on the body shape of 51 patients with Turner's syndrome (aged 6-19 years) was evaluated. Biosynthetic growth hormone was given in a dose of 24 IU/m2 body surface/week for two years. Karyotype analysis on peripheral blood was performed. Patients older than 12 years also received 0.1 microgram ethinyl oestradiol/kg body weight/day orally. Body shape was characterized by studying pairs of measurements expressed as SD scores (z scores). As reference data, our own locally obtained data from normal children were used. After two years of growth hormone therapy, height, sitting height, bi-acromial and bi-iliac diameter increased from -3.7, -2.9, -1.7 and -1.2 to -1.3, -2.5, -0.6 and +0.5 z scores, respectively. The shape of the patients, expressed as height/bi-iliac diameter and also as sitting height/bi-iliac diameter became more abnormal. As no difference could be noted between the prepubertal and pubertal groups or between the XO and mosaic groups, it is suggested that growth hormone treatment causes a relatively wide pelvis in patients with Turner's syndrome.     

Bullous lesions in Henoch Schönlein Purpura as indication to start systemic prednisone

Henoch Schönlein Purpura (HSP) is usually mild and self‐limiting, but it may be accompanied by severe complications such as bullous lesions. We describe the use of systemic prednisone in two patients with bullous lesions in HSP. The first patient presented with progressive bullous lesions distributed on the limbs that evolved into painful ulcers and necrosis. These were further complicated by a secondary skin infection. He then received 1 mg/kg/day prednisone after 9 days. Patient 2, a 10‐year‐old boy, presented with HSP and bullous lesions and received intravenous prednisone 1 mg/kg/day within 48 h after appearance of the bullous lesions. He recovered rapidly without any complications. Conclusion: To reduce the severity of HSP related bullous lesions and their sequelae, we would propose starting prednisone (1 mg/kg/day) as soon as the bullae appear. In addition to prednisone, analgesics and specialist skin care for bullae should be started.