American journal of hematology
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Letter Randomized Controlled Trial Multicenter Study
Beneficial effects of nitric oxide breathing in adult patients with sickle cell crisis.
Pain from vaso-occlusive crisis (VOC) is the major cause of hospitalization in patients with sickle cell disease (SCD). The beneficial therapeutic effects of inhaled nitric oxide (NO) on the pathophysiology of SCD have been reported. A double-blind, randomized, placebo-controlled clinical trial was conducted to determine whether NO breathing reduces acute VOC pain in adult patients and to study the safety of inhaled NO. ⋯ Parenteral morphine use was lower in the inhaled NO group, but the difference was not statistically significant. Safety assessments included systolic blood pressure measurements,pulse oximetry readings, concentration of delivered nitrogen dioxide, and concentration of methemoglobin (metHb). None of these NO toxicities was observed.
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Multiple myeloma is clinically heterogeneous and risk stratification is vital for prognostication and informing treatment decisions. As bortezomib is able to overcome several high-risk features of myeloma, the validity of conventional risk-stratification and prognostication systems needs to be reevaluated. We study the survival data of 261 previously untreated myeloma patients managed at our institution, where bortezomib became available from 2004 for the treatment of relapse disease. ⋯ However, when stratified by bortezomib exposure, only metaphase karyotyping was still discriminating of long-term prognosis. The presence of an abnormal nonhyperdiploid karyotype overrides all other clinical and laboratory parameters in predicting for a worse outcome on multivariate analysis (median survival 2.6 years, P = 0.001), suggesting that bortezomib used at relapse is better able to overcome adverse risk related to high tumor burden (as measured by the ISS) than adverse cytogenetics on conventional karyotyping. Metaphase karyotyping provides additional prognostic information on tumor kinetics where the presence of a normal diploid karyotype in the absence of any high-risk FISH markers correlated with superior survival and could act as a surrogate for lower plasma cell proliferation.
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In a cohort of children with sickle cell disease (SCD) and vaso-occlusive pain visits served through South Carolina's Medicaid system over a 6-year period (N 5 523), we compared the number of vaso-occlusive pain or acute chest syndrome (ACS)/pneumonia episodes, and outpatient or acute service costs in those treated or not treated with hydroxyurea (HU). HU may be an underused intervention for SCD in this practice setting, for a variety of reasons. ⋯ Compared with the non-HU cohort, the HU group evinced a significantly higher risk of experiencing vaso-occlusive pain episodes (RR 5 3.32, P < 0.0001)and ACS/pneumonia episodes (RR 5 2.66, P < 0.0001), and higher outpatient,inpatient/emergency, and total service costs (RR 5 1.85, 2.11,2.10, and P < 0.0001, respectively) over time. HU is clinically effective in reducing pain episodes, hospitalizations, and total care costs, but those receiving it might be more severely ill.
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It is estimated that there are 100,000 people living with sickle-cell disease (SCD) in the United States [1]. The most common manifestation of SCD is vaso-occlusive crisis, which is characterized by intermittent, unexpected episodes of excruciating pain. As these episodes often come on suddenly, much of the care for these crises occurs within emergency departments (EDs). ⋯ For example, in 1997, Woods et al. [2] found that 85.7% of 7,202 hospital admissions for SCD in Illinois were for patients that came through the ED, and the total charges for sickle-cell admissions in Illinois were found to be $30 million a year. A recent study of healthcare use by children with SCD demonstrated that children insured by Medicaid had higher ED utilization than those with private insurance (57% vs.45%) [5]. The purpose of our study was to provide national level estimates of ED utilization by SCD patients, which have not previously been available.
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There is no instrument to measure severity of sickle cell disease (SCD) in pediatric patients that is generally accepted. The aim of this study was to develop and validate a severity index for SCD in children. We developed an index consisting of 12 items and tested its validity of the index using data from 92 children. ⋯ Age and the risk of death score were weakly associated with the pediatric severity index for SCD. This is the first pediatric SCD severity index that was developed and validated using modern clinimetric methodology. The validity and reliability of this index should be further evaluated in a prospective study including a larger cohort, preferably diagnosed at birth.