Initial development of a questionnaire evaluating perceived benefits and barriers to pediatric clinical trials participation

ObjectiveTo evaluate perceived benefits and barriers to pediatric clinical trials participation to improve decision-making and enhance recruitment and retention among minority youth with chronic health conditions (sickle cell disease, asthma) and their caregivers.MethodsA questionnaire was developed based on the social ecological model using input from medical experts and community-based public health organizations. Parallel caregiver, adolescent/young adult (AYA; 16–39 years old), and child (8–15 years old) versions were field tested. Patients and caregivers completed the questionnaire, which included demographic items, perceived life stress and social desirability measures.ResultsExploratory factor analysis rendered a four-factor solution for the caregiver version (direct treatment benefit, mistrust of research/researchers, trust in healthcare team to engage in safe research, and opportunity cost) and the AYA version (mistrust/no direct benefit, safety, direct treatment benefit/practical considerations, and social support for research). Factor structures differed for SCD and asthma caregivers; results were equivocal for the child version. Summated subscales were not significantly associated with patient demographics or social desirability, but significant correlations with perceived life stress and prior participation in research were identified.ConclusionsWhile the factor structure should be confirmed with larger samples, findings indicate potential benefit, perceived harm due to mistrust of researchers, and logistics are primary factors in decision-making about participation in pediatric clinical trials. By addressing these benefits/barriers through adjustments to recruitment and informed consent procedures, researchers may address misperceptions of research, improve decision-making, and increase recruitment and retention particularly for ethnic minority children with chronic health conditions.     

Factor V Leiden, prothrombin 20210G>A, MTHFR 677C>T and 1298A>C, and homocysteinemia in Tunisian blood donors

Specific genetic conditions are known to be associated with high risk of venous thromboembolism. This genetic basis varies widely between ethnic groups. We investigated the distribution of four inherited polymorphisms in 113 unselected Tunisian blood donors by using polymerase chain reaction-restriction fragment length polymorphism (PCR-RFLP) method. The allele frequencies of Factor V Leiden (FVL), prothrombin 20210G>A, methylenetetrahydrofolate reductase (MTHFR) 677C>T, and MTHFR 1298A>C mutations were 3, 0.9, 30, and 31%, respectively. The MTHFR 677C>T polymorphism was influenced by age. Twenty-nine of the 113 blood donors demonstrated more than one genetic markers. Hyperhomocysteinemia was found in 12 subjects, and it was statistically associated to the MTHFR 677TT genotype. Principal component analysis allowed disclosing the resemblance between Mediterranean populations. Our findings may be helpful for population genetics study, and provide epidemiologic database for further studies in thrombosis field among Tunisians.     

MRI is it complementary or mandatory to ultrasound in classification of different congenital anomalies of female reproductive tract?

Aim of work

To assess the added value of MRI compared to US in diagnosis of Mullerian duct anomalies and its subtypes thus guiding proper management plans.

Role of diffusion tensor imaging in carpal tunnel syndrome: A case control comparative study to electrophysiological tests and clinical assessment

Purpose

To study the efficacy of diffusion tensor imaging (DTI) in the diagnosis of carpal tunnel syndrome (CTS).

Single autologous stem-cell transplantation followed by maintenance therapy with thalidomide is superior to double autologous transplantation in multiple myeloma: results of a multicenter randomized clinical trial

From April 2003 to December 2006, 195 patients with de novo symptomatic myeloma and younger than 60 years of age were randomly assigned to receive either tandem transplantation up front (arm A, n = 97) or one autologous stem-cell transplantation followed by a maintenance therapy with thalidomide (day + 90, 100 mg per day during 6 months) (arm B, n = 98). Patients included in arm B received a second transplant at disease progression. In both arms, autologous stem-cell transplantation was preceded by first-line therapy with thalidomide-dexamethasone and subsequent collection of peripheral blood stem cells with high-dose cyclophosphamide (4 g/m(2)) and granulocyte colony stimulating factor. Data were analyzed on an intent-to-treat basis. With a median follow-up of 33 months (range, 6-46 months), the 3-year overall survival was 65% in arm A and 85% in arm B (P = .04). The 3-year progression-free survival was 57% in arm A and 85% in arm B (P = .02). Up-front single autologous transplantation followed by 6 months of maintenance therapy with thalidomide (with second transplant in reserve for relapse or progression) is an effective therapeutic strategy to treat multiple myeloma patients and appears superior to tandem transplant in this setting. This study was registered at www.ClinicalTrials.gov as (NCT 00207805).     

An escort for GPCRs: implications for regulation of receptor density at the cell surface

G-protein-coupled receptors (GPCRs) are dynamically regulated by various mechanisms that tune their response to external stimuli. Modulation of their plasma membrane density, via trafficking between subcellular compartments, constitutes an important process in this context. Substantial information has been accumulated on cellular pathways that remove GPCRs from the cell surface for subsequent degradation or recycling. In comparison, much less is known about the mechanisms controlling trafficking of neo-synthesized GPCRs from intracellular compartments to the cell surface. Although GPCR export to the plasma membrane is commonly considered to mostly implicate the default, unregulated secretory pathway, an increasing number of observations indicate that trafficking to the plasma membrane from the endoplasmic reticulum might be tightly regulated and involve specific protein partners. Moreover, a new paradigm is emerging in some cellular contexts, in which stocks of functional receptors retained within intracellular compartments can be rapidly mobilized to the plasma membrane to maintain sustained physiological responsiveness.