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Background and Objective. Hematopoietic stem cell transplantation (HSCT) in developing countries is cost-limited. Our primary goal was to determine the cost structure for the HSCT program model developed over the last decade at our public university hospital and to assess its clinical outcomes. Materials and Methods. Adults and children receiving an allogeneic hematopoietic stem cell transplant from January 2010 to February 2011 at our hematology regional reference center were included. Laboratory tests, medical procedures, chemotherapy drugs, other drugs, and hospitalization costs were scrutinized to calculate the total cost for each patient and the median cost for the procedure. Data regarding clinical evolution were incorporated into the analysis. Physician fees are not charged at the institution and therefore were not included. Results. Fifty patients were evaluated over a 1-year period. The total estimated cost for an allogeneic HSCT was $12,504. The two most expensive diseases to allograft were non-Hodgkin lymphoma ($11,760 ± $2,236) for the malignant group and thalassemia ($12,915 ± $5,170) for the nonmalignant group. Acute lymphoblastic leukemia ($11,053 ± 2,817) and acute myeloblastic leukemia ($10,251 ± $1,538) were the most frequent indications for HSCT, with 11 cases each. Median out-of-pocket expenses were $1,605, and 1-year follow-up costs amounted to $1,640, adding up to a total cost of $15,749 for the first year. The most expensive components were drugs and laboratory tests. Conclusion. Applying the cost structure described, HSCT is an affordable option for hematological patients living in a developing country. [ABSTRACT FROM AUTHOR]

Objective. The aim of this study was to determine the short- and long-term efficacy and safety of 8-weekly canakinumab therapy in children with cryopyrin-associated periodic syndromes (CAPS) in routine clinical practice.Methods. A single-centre observational study was performed. Patients were assessed every 8 weeks at a dedicated clinic. Standardized assessments were the 10-domains DAS for CAPS, acute phase reactants (APRs), physician’s global assessment of disease activity, Child Health Assessment Questionnaire (CHAQ) and Child Health Questionnaire Parent Form 28 (CHQPF-28). The primary endpoint was clinical improvement, defined as a reduction of DAS score 8 weeks after commencing therapy. Secondary endpoints included sustained clinical improvement in APRs, relapses, CHAQ score and CHQPF-28 score.Results. Ten children with CAPS [eight Muckle-Wells syndrome (MWS), two chronic infantile cutaneous neurological articular (CINCA); median age 6.3 years] received 8-weekly canakinumab treatments at 2–8.7 mg/kg for a median of 21 months (range 12–31 months). Nine of 10 patients improved after the first dose: baseline median DAS of 7.5/20 decreased to 3.5/20 at 8 weeks (P = 0.04). This clinical improvement was sustained at a median follow-up of 21 months (range 12–31 months). Children with CINCA required higher doses of canakinumab than those with MWS. CHAQ and CHQ scores indicated improvement in functioning and health-related quality of life (HRQoL). Treatment was well tolerated, with no injection site reactions and no serious infections.Conclusion. Canakinumab, although costly, is a safe and effective treatment for CAPS in children, leading to sustained improvement in disease activity, serological markers, functional ability and HRQoL. [ABSTRACT FROM PUBLISHER]

Purpose. Patient safety and cost-control benefits achieved through a medical center's conversion to commercial fluids for continuous renal-replacement therapy (CRRT) are described. Summary. In keeping with national patient safety recommendations, a medical center undertook a quality-improvement initiative to increase the standardization of CRRT fluids and associated patient care practices. After a feasibility study to ascertain CRRT fluid usage patterns and baseline costs, an appropriate commercial product was selected. The transition was implemented over several months in a three-phase process entailing (1) conversion to a commercial product for regional citrate anticoagulation, (2) changing the standard formula on preprinted orders to one that modeled a commercial product, and (3) conversion to the actual commercial product as the standard. Thorough education of all parties involved, as well as modifications to preprinted orders, occurred during each phase of the project. The use of the commercial product reduced the need for pharmacy compounding of CRRT fluids by more than 80%. A postconversion cost analysis indicated sharp reductions of product and labor (i.e., compounding) expenses relative to estimated baseline costs, with estimated total savings of at least $399,290 during the first six months after the conversion initiative. Implementation challenges included the need to manually adjust the potassium content of the commercial CRRT fluid and the need to provide i.v. supplementation of phosphorus and magnesium to some patients receiving the fluid. The initiative emphasized the need for institution-specific research and planning before conversion initiatives targeting CRRT fluids and other critical therapies, as well as proper education and training of all parties involved. Conclusion. The standardization of CRRT fluids using commercially available products improved a medical center's compliance with patient safety standards and yielded substantial cost savings. [ABSTRACT FROM AUTHOR]