Stem cell transplantation for patients with Fanconi anemia with low-dose cyclophosphamide and antithymocyte globulins without the use of radiation therapy

In all, 22 patients with confirmed Fanconi anemia (FA) underwent stem cell transplantation (SCT) from HLA-matched, related donors at KFSHRC. Median age at SCT was 7.6 years (range, 2.5-14.6 years). Conditioning regimen consisted of cyclophosphamide (CY) 15 mg/kg/day intravenously (i.v.) for 4 consecutive days, in addition to equine antithymocyte globulins (ATG) given i.v. at 40 mg/kg/day for four doses pre-SCT. No radiation therapy was given. For graft-versus-host disease prophylaxis, we used cyclosporin at the standard doses; ATG was added at 20 mg/kg/dose i.v. on days 2, 4, 6, 8, 10, and 12 post-SCT (total of six doses). All patients engrafted and are alive and transfusion independent with a median follow-up time of 20.2 months (range, 3.3-59 months). One patient however developed a decrease in her WBC and platelet count. Her work-up revealed slightly hypocellular bone marrow, and a series of chimerism studies over 1 year confirmed that she has stable mixed chimerism; she remains transfusion independent. We conclude that low-dose CY without radiation therapy can be used satisfactorily in the conditioning of patients with FA undergoing related SCT.     

A community-based study of early childhood sensory stimulation in home environment associated with growth and psychomotor development in Pakistan

Objectives

Sensory stimulation (SS) is a non-nutritional modifiable risk factor for early childhood development. We assessed SS in home environment and examined its influence on physical growth and psychomotor development (PD).

Methods

A cross-sectional study was conducted in 26 communities in Pakistan among children aged <3 (n = 1,219). They were assessed at home visits using (1) Bayley’s Infant Developmental Scale for PD, (2) home observation for measurement of the environment inventory for SS, (3) anthropometry and (4) socio-economic questionnaire.

Results

In rural homes, SS provided was lower as compared to urban counterparts (Adj mean diff: 4.47, 95 % CI 3.78, 5.16) and showed an association with stunting (Adj mean diff: ?1.30, 95 % CI ?1.93, ?0.66), and underweight (Adj mean diff: ?1.04, 95 % CI ?1.71, ?0.38) not explained by type of neighbourhood or socio-economic status. SS was associated with PD more than combined contribution of socio-economic status and rural–urban factors (Adj mean diff: 0.47, 95 % CI 0.30, 0.63).

Conclusions

SS in rural homes may be a significant factor influencing the child development. There is a need to corroborate these results by additional research for integration in health policy initiatives.     

Fetoscopic approach to amniotic band syndrome

Background

Amniotic band syndrome (ABS) is an uncommon complication of pregnancy that can result in fetal demise.

Methods

We present our experience with fetoscopic amniotic band release.

Results

Five patients underwent fetoscopic amniotic band release for preoperatively diagnosed ABS involving at least one extremity. Four of five patients were found to have involvement of the umbilical cord at the time of fetoscopy. One of these four did not have the band released and underwent fetal demise at 24 weeks. All four survivors had good functional outcomes of affected limbs. Two patients developed membrane separation and had preterm deliveries at 32 weeks gestation whereas the other two carried to term. No maternal complications were noted.

Conclusions

Fetoscopic amniotic band release is safe. Umbilical cord involvement is difficult to assess preoperatively, but when it is present should be treated to reduce the risk of fetal demise.     

Sustained release and enhanced oral bioavailability of rivaroxaban by PLGA nanoparticles with no food effect

Journal of Thrombosis and Thrombolysis - The purpose of the currents study was to enhance bioavailability of rivaroxaban (RXB) and reduce the food effect. RXB loaded PLGA nanoparticles...     

Small Cell Neuroendocrine Carcinoma of the Oropharynx Harbouring Oncogenic HPV-Infection

Small cell carcinoma/neuroendocrine carcinoma (SCNEC) of the oropharynx is uncommon. Recently, an association has been reported between oropharyngeal SCNEC and high-risk human papillomavirus (HPV) infection. While HPV infection confers a better prognosis for oropharyngeal squamous cell carcinoma, HPV infection does not appear to influence the biological behaviour of SCNECs, which are generally associated with poor clinical outcomes. We document two cases of SCNEC arising in the oropharynx with evidence of high-risk HPV infection. The cases highlight the expanding range of malignant oropharyngeal neoplasms that harbour oncogenic HPV infection and support the concept that, irrespective of HPV infection, neuroendocrine differentiation portends a poor prognosis.     

Immunological mechanisms underpinning faecal microbiota transplantation for the treatment of inflammatory bowel disease

Inflammatory bowel disease (IBD) is a chronic gastrointestinal disease that results from a dysregulated immune response against specific environmental triggers in a genetically predisposed individual. Increasing evidence has indicated a causal role for changes in gut microbiota (dysbiosis) contributing to this immune-mediated intestinal inflammation. These mechanisms involve dysregulation of multiple facets of the host immune pathways that are potentially reversible. Faecal microbiota transplantation (FMT) is the transfer of processed stool from a healthy donor into an individual with an illness. FMT has shown promising results in both animal model experiments and clinical studies in IBD in the resolution of intestinal inflammation. The underlying mechanisms, however, are unclear. Insights from these studies have shown interactions between modulation of dysbiosis via changes in abundances of specific members of the gut microbial community and changes in host immunological pathways. Unravelling these causal relationships has promising potential for a translational therapy role to develop targeted microbial therapies and understand the mechanisms that underpin IBD aetiopathogenesis. In this review, we discuss current evidence for the contribution of gut microbiota in the disruption of intestinal immune homeostasis and immunoregulatory mechanisms that are associated with the resolution of inflammation through FMT in IBD.     

Pilot study protocol of a mHealth self-management intervention for family members of pediatric transplant recipients

Solid-organ transplantation is the treatment of choice for end-stage organ failure. Parents of pediatric transplant recipients who reported a lack of readiness for discharge had more difficulty coping and managing their child's medically complex care at home. In this paper, we describe the protocol for the pilot study of a mHealth intervention (myFAMI). The myFAMI intervention is based on the Individual and Family Self-Management Theory and focuses on family self-management of pediatric transplant recipients at home. The purpose of the pilot study is to test the feasibility of the myFAMI intervention with family members of pediatric transplant recipients and to test the preliminary efficacy on postdischarge coping through a randomized controlled trial. The sample will include 40 family units, 20 in each arm of the study, from three pediatric transplant centers in the United States. Results from this study may advance nursing science by providing insight for the use of mHealth to facilitate patient/family–nurse communication and family self-management behaviors for family members of pediatric transplant recipients.     

Correction: Acridinedione as selective flouride ion chemosensor: a detailed spectroscopic and quantum mechanical investigation

Correction for ‘Acridinedione as selective flouride ion chemosensor: a detailed spectroscopic and quantum mechanical investigation’ by Nafees Iqbal et al., RSC Adv., 2018, 8, 1993–2003.

The authors regret that the interpretation of the fluorescence spectra of compound 7i published in the original article was incorrect. In the original article, it was reported that upon excitation at 380 nm, the fluorescence spectrum of compound 7i showed two emission bands at 450 nm and 770 nm (Fig. 5b of the original article). The signal at 770 nm (previously reported as an emission band), is instead a second order diffraction (an artefact of diffraction grating/spectrofluorometer monochromator), as revealed from the literature.^1,2 The authors thank a reader for highlighting this mistake.The Royal Society of Chemistry apologises for these errors and any consequent inconvenience to authors and readers.     

Correction: Development and in vitro evaluation of κ-carrageenan based polymeric hybrid nanocomposite scaffolds for bone tissue engineering

Correction for ‘Development and in vitro evaluation of κ-carrageenan based polymeric hybrid nanocomposite scaffolds for bone tissue engineering’ by Muhammad Umar Aslam Khan et al., RSC Adv., 2020, 10, 40529–40542. DOI: 10.1039/D0RA07446B.

The authors regret errors in Fig. 9 in the original article. The corrected Fig. 9 is shown below where all three +ive control panels and the 72 h CG-g-Aac-2 panel have been replaced.Open in a separate windowFig. 9Cell morphology of MC3T3-E1 against +ive control and all scaffold samples (CG-g-AAc1, CG-g-AAc2 and CG-g-AAc3) under standard in vitro conditions. The red arrows show thread-like morphology and the yellow arrows exhibits well-grown morphology of the cells.The Royal Society of Chemistry apologises for these errors and any consequent inconvenience to authors and readers.