Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study

We conducted a multicenter pilot investigation of the safety and feasibility of bone marrow transplantation (BMT) in adults with severe sickle cell disease (SCD) (NCT 01565616) using a reduced toxicity preparative regimen of busulfan (13.2 mg/kg), fludarabine (175 mg/m²) and thymoglobulin (6 mg/kg) and cyclosporine or tacrolimus and methotrexate for graft-vs-host disease (GVHD) prophylaxis. Twenty-two patients (median age 22 years; range 17-36) were enrolled at eight centers. Seventeen patients received marrow from an HLA-identical sibling donor and five patients received marrow from an 8/8 HLA-allele matched unrelated donor. Before BMT, patients had stroke, acute chest syndrome, recurrent pain events, were receiving regular red blood cell transfusions, or had an elevated tricuspid regurgitant jet (TRJ) velocity, which fulfilled eligibility criteria. Four patients developed grades II-III acute GVHD (18%) and six developed chronic GVHD (27%) that was moderate in two and severe in one patient. One patient died of intracranial hemorrhage and one of GVHD. Nineteen patients had stable donor chimerism, 1-year post-transplant. One patient who developed secondary graft failure survives disease-free after a second BMT. The one-year overall survival and event-free survival (EFS) are 91% (95% CI 68%-98%) and 86% (95% CI, 63%-95%), respectively, and 3-year EFS is 82%. Statistically significant improvements in the pain interference and physical function domains of health-related quality of life were observed. The study satisfied the primary endpoint of 1-year EFS ≥70%. This regimen is being studied in a prospective clinical trial comparing HLA-matched donor BMT with standard of care in adults with severe SCD (NCT02766465).     

31P MR spectroscopic imaging detects regenerative changes in human liver stimulated by portal vein embolization

Purpose:

First, to evaluate hepatocyte phospholipid metabolism and energetics during liver regeneration stimulated by portal vein embolization (PVE) using proton‐decoupled ³¹P MR spectroscopic imaging (³¹P‐MRSI). Second, to compare the biophysiologic differences between hepatic regeneration stimulated by PVE and by partial hepatectomy (PH).

Materials and Methods:

Subjects included six patients with hepatic metastases from colorectal cancer who were scheduled to undergo right PVE before definitive resection of right‐sided tumor.³¹P‐MRSI was performed on the left liver lobe before PVE and 48 h following PVE. Normalized quantities of phosphorus‐containing hepatic metabolites were analyzed from both visits. In addition, MRSI data at 48 h following partial hepatectomy were compared with the data from the PVE patients.

Results:

At 48 h after PVE, the ratio of phosphomonoesters to phosphodiesters in the nonembolized lobe was significantly elevated. No significant changes were found in nucleoside triphosphates (NTP) and Pi values. The phosphomonoester (PME) to phosphodiester (PDE) ratio in regenerating liver 48 h after partial hepatectomy was significantly greater than PME/PDE 48 h after PVE.

Conclusion:

³¹P‐MRSI is a valid technique to noninvasively evaluate cell membrane metabolism following PVE. The different degree of biochemical change between partial hepatectomy and PVE indicates that hepatic growth following these two procedures does not follow the same course. J. Magn. Reson. Imaging 2011;. © 2011 Wiley‐Liss, Inc.     

The sound of operation and the acoustic attenuation of the Ohmeda Medical Giraffe OmniBed™

The neonatal intensive care unit (NICU) is an environment that provides premature and fragile infants with health provisions needed to make a complete recovery. Premature infants are often born before their auditory systems have had an opportunity to fully mature. Research has shown that the ambient acoustic environment in the NICU exceeds the maximum noise level recommended by the American Academy of Pediatrics, even after measures have been taken to decrease noise levels. The purpose of this study is to evaluate noise levels inside an Ohmeda Medical Giraffe? OmniBed?, the natural attenuation of the incubator, and the effects of modifications on attenuation and reverberation within the Giraffe TM OmniBed?. The normal operation of the Giraffe? OmniBed? is 41.7 dBA which indicates a lower noise of operation than previous studies. The Giraffe? OmniBed? naturally attenuates 12 dBA. Leaving an access latch or portal door open causes a statistically significant (P=.001) increase in sound within the bassinet. All modifications in the no-noise and the noise conditions showed a statistically significant (P=.001) drop in L(eq) when compared to baseline.     

Using a qualitative approach to develop an evaluation data set for community-based health promotion programs addressing racial and ethnic health disparities

Racial and ethnic disparities in health have increasingly become a central focus of health promotion efforts. At the community level, however, collecting data and evaluating these programs has been a challenge because of the diversity of populations, community contexts, and health issues as well as a range of capacities for conducting evaluation. This article outlines a qualitative research process used to develop a Web-based standard program performance data reporting system for programs funded by the U.S. Office of Minority Health (OMH), but generally applicable to community-based health promotion programs addressing health disparities. The "core-and-module" data set, known as the Uniform Data Set (UDS), is a Web-based system and is used as the programwide reporting system for OMH. The process for developing the UDS can be used by any agency, locality, or organization to develop a tailored data collection system allowing comparison across projects via an activity-based typology around which data reporting is structured. The UDS model enables the collection of grounded data reflecting community-level steps necessary to address disparities as well as a reporting structure that can guide data collection based on broader frameworks now emerging that specify criteria for measuring progress toward the elimination of health disparities.     

Child coping and parent coping assistance during the peritrauma period in injured children

Pediatric physical injury is a very common, potentially traumatic medical event that many families face each year. The role that child or parent coping behavior plays in emotional recovery from injury is not well understood. This study described coping used by children and coping assistance implemented by parents in the early aftermath of a child's injury. Ten child-parent dyads participated in individual semistructured interviews that were audiorecorded, transcribed, and coded using hierarchical coding schemes. Study findings highlight reliance on a broad range of coping strategies. Although children and parents report some similarities in their perceptions of child coping, parents do not recognize all the coping strategies that children report. This suggests potential for improvement in parent-child communication concerning coping techniques. Parents report a limited number of coping assistance strategies, indicating a niche for preventive programs. Further research should examine coping during the peritrauma period as it relates to physical and emotional outcomes to inform secondary prevention programs.