Background
Although hematopoietic stem cell transplantation (HSCT) is the treatment of choice for childhood myelodysplastic syndrome (MDS), there is no consensus regarding patient or disease characteristics that predict outcomes.
Procedure
We reviewed 37 consecutive pediatric MDS patients who received myeloablative HSCT between 1990 and 2010 at a single center.
Results
Twenty had primary MDS and 17 had secondary MDS. Diagnostic cytogenetics included monosomy 7 (n=21), trisomy 8 (n=7) or normal/other (n=8). According to the modified WHO MDS classification, thirty had refractory cytopenia and 7 had refractory anemia with excess blasts. IPSS scores were: low risk (n=1), intermediate-1 (n=15), and intermediate-2 (n=21). OS and DFS at 10-years in the entire cohort was 53% and 45%. Relapse at 10-years was 26% and 1-year TRM was 25%. In multivariate analysis, factors associated with improved 3-year DFS were not receiving pre-HSCT chemotherapy (RR=0.30, 95% CI 0.10–0.88; p=0.03) and a shorter interval (<140 days) from time of diagnosis to transplant (RR=0.27, 95% CI 0.09–0.80; p=0.02). 3-year DFS in patients who did not receive pre-HSCT chemotherapy and those who had a shorter interval to transplant (n=16) was 80%.
Conclusion
These results suggest that children with MDS should be referred for allogeneic HSCT soon after diagnosis and that pre-HSCT chemotherapy does not appear to improve outcomes.
Background-Transfer for primary percutaneous coronary intervention (PCI) is superior to fibrinolysis if performed in a timely manner but frequently requires dislocation of patients and their families from their local community. Although patient satisfaction is increasingly viewed as an important quality indicator, there are no data on how emergent transfer for PCI affects patients with ST-segment-elevation myocardial infarction and their families. Methods and Results-The Minneapolis Heart Institute's Level 1 Regional ST-Segment-Elevation Myocardial Infarction program is designed to facilitate emergent transfer for PCI in patients with ST-segment-elevation myocardial infarction from 31 rural and community hospitals. To determine the effect of emergent transfer, questionnaires were given to 152 patients and their families who survived to hospital discharge with a 65.8% response rate (mean age, 63.9 years; 29% women). Ninety-five percent of patients felt the reasons and process of transfer were well explained, and 97% felt transfer for care was necessary. Despite this, 15% of patients would have preferred to stay in their local hospital. The majority of the families felt the transfer process (88%) and family member's condition (94%) were well explained. Although 99% felt it was necessary for their family member to be transferred for specialized care, 11% of families still would have preferred that their family members remain at the local community hospital. Conclusions-Our results suggest that ST-segment-elevation myocardial infarction patients and families can be informed, even in time-critical situations, about the transfer process for PCI and understand the need for specialized care. Still, a significant minority would prefer to stay at their local hospital, despite acknowledging transfer for PCI provided optimal care. (Circ Cardiovasc Qual Outcomes. 2014;7:244-250.)Key Words: patient education as topic ◼ quality of health care ◼ ST-elevation myocardial infarction ◼ systems of care
Langerhans cell histiocytosis (LCH) is a proliferative disorder of dendritic cells which has evaded efforts to clearly define pathogenesis, diagnostic criteria, and therapeutic response markers. Strides have been made at classification with the recent development of a quantified score of disease severity. Splenic involvement is an indicator of poor prognosis, with spleen size its surrogate marker in evaluation and scoring. We describe a case of pediatric LCH with progressive splenomegaly despite treatment, which when examined at splenectomy revealed no LCH involvement but extramedullary hematopoiesis. These findings thus challenge our understanding of splenomegaly as a marker of disease.
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