rss_2.0Pharmacy FeedSciendo RSS Feed for Pharmacy Feed flavanone inhibits cell viability in PC-3 human prostate cancer by inducing cellular apoptosis, ROS production and cell cycle arrest<abstract><title style='display:none'>Abstract</title><p>The main purpose of the present study was to evaluate the antitumor effects of pinocembrin in human prostate cancer cells (PC-3) along with investigating its effects on cell apoptosis, endogenous ROS production and cell cycle. MTT assay and clonogenic assays were used to study the effects on cell viability and cancer colony formation, respectively. Fluorescence microscopy along with Western blotting was used to study apoptotic effects induced by pinocembrin. Flow cytometry was used to study effects on ROS production and cell cycle phase distribution. Results indicated that pinocembrin promoted inhibition cell proliferation along with reducing cancer colony formation of PC-3 cells in a dose-dependent manner. Pinocembrin induced regulatory effects over expressions of caspase-3, caspase-9, Bax and Bcl-2, thereby promoting apoptotic cell death in PC-3 cells. It also led to the dose-dependent G0/G1 cell cycle arrest. In conclusion, pinocembrin exhibits strong anticancer effects in human prostate cancer cells mediated <italic>via</italic> apoptosis, endogenous ROS production and G0/G1 cell cycle arrest.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Recent advancements to enhance the therapeutic efficacy of antiepileptic drugs<abstract><title style='display:none'>Abstract</title><p>Epilepsy is a multifactorial neurological disorder characterized by recurrent or unprovoked seizures. Over the past two decades, many new antiepileptic drugs (AEDs) were developed and are in use for the treatment of epilepsy. However, drug resistance, drug-drug interaction and adverse events are common problems associated with AEDs. Antiepileptic drugs must be used only if the ratio of efficacy, safety, and tolerability of treatment are favorable and outweigh the disadvantages including treatment costs. The application of novel drug delivery techniques could enhance the efficacy and reduce the toxicity of AEDs. These novel techniques aim to deliver an optimal concentration of the drug more specifically to the seizure focus or foci in the CNS without numerous side-effects. The purpose of this article is to review the recent advancements in antiepileptic treatment and summarize the novel modalities in the route of administration and drug delivery, including gene therapy, for effective treatment of epilepsy.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Antioxidant and antihyperglycemic activities of radical leaves in streptozocin-induced diabetic rats<abstract><title style='display:none'>Abstract</title><p><italic>Scorzonera</italic> species are used for treating various diseases. They are consumed raw, especially in the spring, and have nutritious and dietetic values. This study evaluated the antidiabetic and antioxidant effects of ethanolic extracts of <italic>Scorzonera cinerea</italic> (Sc) radical leaves in diabetes mellitus. Five random groups of Wistar rats (<italic>n</italic> = 8) were created – control, diabetic, acarbose, Sc-Dried, and Sc-Frozen. Phenolic profiles of extracts were determined by HPLC. Free radical scavenging capacity was measured using DPPH and ABTS tests. The inhibitory effects of Sc extracts on α-glucosidase and α-amylase activities were also evaluated. Moreover, superoxide dismutase (SOD), glutathione peroxidase (GPx), and catalase (CAT) activities, glutathione (GSH) concentration, malondialdehyde (MDA), total antioxidant status (TAS) and total oxidant status (TOS) were analyzed in the liver tissues. While dried <italic>Scorzonera</italic> extract was more effective in α-amylase inhibitory activity, frozen <italic>Scorzonera</italic> extract was more effective in α-glucosidase inhibitory activity. Sc-Dried and Sc-Frozen extracts lowered blood glucose and HbA<sub>1c</sub> levels, they also increased insulin. Although liver MDA and TOS were significantly increased in the diabetic group, their values were significantly lower in the Sc-Dried- and Sc-Frozen-treated groups. GSH, TAS, and anti-oxidant enzyme activities decreased in the diabetic group, but Sc-Dried and Sc-Frozen supplements significantly enhanced liver antioxidant values. In conclusion, <italic>S</italic>. <italic>cinerea</italic> treatment exerts potential hypoglycemic and antioxidant effects in diabetes. Thus, it can be considered as a candidate dietary supplement for health benefits in diabetes.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Identification and pharmacokinetics of saponins in after oral administration to rats by HPLC-Q-TOF/MS and HPLC-MS/MS<abstract><title style='display:none'>Abstract</title><p><italic>Rhizoma Anemarrhenae</italic> is a well-known herbal medicine with saponins as its commonly regarded major bioactive components. It is essential to classify the properties of saponins which are associated with their toxicity and efficacy. In this study, 25 compounds were identified by HPLC-Q-TOF/MS in the extract of <italic>Rhizoma Anemarrhenae</italic> and 8 saponins were detected in rat plasma by HPLC-MS/MS after oral administration of this extract. These were neomangiferin, mangiferin, timosaponin E1, timosaponin E, timosaponin B-II, timosaponin B-III, timosaponin A-III and timosaponin A-I. A sensitive and accurate HPLC-MS/MS method was developed and successfully applied to a pharmacokinetic study of the abovementioned eight saponins after oral administration of the <italic>Rhizoma Anemarrhenae</italic> extract to rats. The method validation, including specificity, linearity, precision, accuracy, recovery, matrix effect and robustness, met the requirements of the intended use. The pharmacokinetic parameter, <italic>T</italic><sub>max</sub> value, ranged from 2 to 8 h for these eight saponins whereas their elimination half-life <italic>(t</italic><sub>1/2</sub>) ranged from 4.06 to 9.77 h, indicating slow excretion. The plasma concentrations of these eight saponins were all very low, indicating a relatively low oral bioavailability. All these results provide support for further clinical studies.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Pharmacokinetics study of a supersaturatable self-microemulsifying drug delivery system for ellagic acid by UHPLC-Q-TOF-MS<abstract><title style='display:none'>Abstract</title><p>To evaluate the bioavailability of ellagic acid loaded super-saturatable self-microemulsifying drug delivery system (S-SMEDDS), its pharmacokinetic properties were studied in rats with an ultra-high performance liquid chromatography-quadrupole time-of-flight tandem mass spectrometry. The plasma samples were treated by solid-phase extraction method, and gallic acid was used as the internal standard when determining the concentration of ellagic acid. Results showed that the established analytical method was sensitive and accurate, which is applicable to the pharmacokinetic study of ellagic acid. The drug was found to be absorbed rapidly <italic>in vivo</italic>, and the plasma concentration-time curve showed double peaks, indicating that ellagic acid were reabsorbed by entero-hepatic circulation after oral administration. Compared with ellagic acid suspension, the apparent clearance of ellagic acid-loaded S-SMEDDS and SMEDDS reduced significantly, and the <italic>AUC</italic><sub>0~t</sub> of them were 4.7 and 5.8-fold increase, respectively. Therefore, the bioavailability of ellagic acid-loaded S-SMEDDS was higher than that of the suspension and SMEDDS.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00New derivatives of sulfonylhydrazone as potential antitumor agents: Design, synthesis and cheminformatics evaluation<abstract><title style='display:none'>Abstract</title><p>Phosphoinositide 3-kinase α (PI3Kα) is a propitious target for designing anticancer drugs. A series of new <italic>N</italic>’-(diphenylmethylene)benzenesulfonohydrazide was synthesized and characterized using FT-IR, NMR (<sup>1</sup>H and <sup>13</sup>C), HRMS, and elemental analysis. Target compounds exhibited an antiproliferative effect against the human colon carcinoma (HCT-116) cell line. Our cheminformatics analysis indicated that the <italic>para</italic>-tailored derivatives [<italic>p</italic>-NO<sub>2</sub> (<bold>3</bold>) and <italic>p</italic>-CF<sub>3</sub> (<bold>7</bold>)] have better ionization potentials based on calculated Moran autocorrelations and ionization potentials. Subsequent <italic>in vitro</italic> cell proliferation assays validated our cheminformatics results by providing experimental evidence that both derivatives <bold>3</bold> and <bold>7</bold> exhibited improved antiproliferative activities against HCT-116. Hence, our results emphasized the importance of electron-withdrawing groups and hydrogen bond-acceptors in the rational design of small-molecule chemical ligands targeting PI3Kα. These results agreed with the induced-fit docking against PI3Kα, highlighting the role of <italic>p</italic>-substituted aromatic rings in guiding the ligand-PI3Kα complex formation, by targeting a hydrophobic pocket in the ligand-binding site and forming π-stacking interactions with a nearby tryptophan residue.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Quality-by-design in pharmaceutical development: From current perspectives to practical applications<abstract><title style='display:none'>Abstract</title><p>Current pharmaceutical research directions tend to follow a systematic approach in the field of applied research and development. The concept of quality-by-design (QbD) has been the focus of the current progress of pharmaceutical sciences. It is based on, but not limited, to risk assessment, design of experiments and other computational methods and process analytical technology. These tools offer a well-organized methodology, both to identify and analyse the hazards that should be handled as critical, and are therefore applicable in the control strategy. Once implemented, the QbD approach will augment the comprehension of experts concerning the developed analytical technique or manufacturing process. The main activities are oriented towards the identification of the quality target product profiles, along with the critical quality attributes, the risk management of these and their analysis through <italic>in silico</italic> aided methods. This review aims to offer an overview of the current standpoints and general applications of QbD methods in pharmaceutical development.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Piperazine sulfonamides as DPP-IV inhibitors: Synthesis, induced-fit docking and biological evaluation<abstract><title style='display:none'>Abstract</title><p>Diabetes mellitus is a chronic illness that needs persistent medical attention and continuous patient self-management to avoid acute complications. Dipeptidyl peptidase-IV (DPP-IV) inhibitors minimize glucagon and blood glucose levels by increasing the incretin levels, glucagon-like peptide (GLP-1) and glucose-dependent insulinotropic poly-peptide (GIP), leading to insulin secretion from pancreatic beta cells. In the present study, nine 1,4-bis(phenylsulfonyl) piperazine derivatives <bold>1a</bold>-<bold>i</bold> were synthesized and identified using <sup>1</sup>H NMR, <sup>13</sup>C NMR, MS and IR spectroscopies. These compounds were tested <italic>in vitro</italic> and showed inhibitory activity ranging from 11.2 to 22.6 % at 100 µmol L<sup>–1</sup> concentration. Piperazine sulfonamide derivatives were found to be promising DPP-IV inhibitors, where the presence of electron-withdrawing groups such as Cl (<bold>1a</bold>-<bold>c</bold>) improved the activity of the compounds more than electron-donating groups such as CH<sub>3 (</sub><bold>1d</bold>-<bold>f</bold>) at the same position. Additionally, <italic>meta</italic>-substitution is disfavored (<bold>1b</bold>, <bold>1e</bold>, <bold>1g</bold>). Induced-fit docking studies suggested that the targeted compounds <bold>1a</bold>-<bold>i</bold> occupy the binding domain of DPP-IV and form H-bonding with the backbones of R125, E205, E206, F357, K554, W629, Y631, Y662 and R669.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Anticancer effects of 7,8-dihydromethysticin in human leukemia cells are mediated cell-cycle dysregulation, inhibition of cell migration and invasion and targeting JAK/STAT pathway<abstract><title style='display:none'>Abstract</title><p>The main focus of this research work was to study the anti-cancer properties of 7,8-dihydromethysticin against HL-60 leukemia cells. Investigations were also performed to check its impact on the phases of the cell cycle, cell migration and invasion, JAK/STAT signalling pathway and intracellular mitochondrial membrane potential (MMP) and reactive oxygen species (ROS). Cell proliferation was assessed through 3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyl tetrazolium bromide (MTT) assay and effects on colony formation were examined <italic>via</italic> clonogenic assay. Flow cytometry and Western blott analysis were performed to investigate the distribution of cell cycle phases. Flow cytometric analysis was performed for the examination of MMP and ROS production. The effect on JAK/STAT signalling pathway was examined through Western blot analysis. Results depicted that 7,8-dihydromethysticin induced concentration- as well as time-dependent inhibition of cell proliferation in leukemia HL-60 cells. Clonogenic assay indicated potential suppression in leukemia HL-60 cell colonies. The 7,8-dihydromethysticin molecule also caused cell cycle arrest at G2/M-phase along with concentration-dependent inhibition of cyclin B1, D1 and E. ROS and MMP measurements indicated significant ROS enhancement and MMP suppression with increasing 7,8-dihydromethysticin concentrations. Additionally, 7,8-dihydromethysticin led to remarkable dose-reliant inhibition of cell invasion as well as cell migration. Therefore, 7,8-dihydromethysticin should be considered a valuable candidate for leukemia research and chemoprevention.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Propolis – quality analysis and use in topical formulations<abstract><title style='display:none'>Abstract</title><p>The aim of this study was to produce propolis extracts, assess their quality and effect on skin cells and determine the penetration of active ingredients from designed semi-solid topical formulations. The use of higher-concentration ethanol and a larger amount of raw material allows extracting a larger quantity of active ingredients from raw propolis. Ultrasound extraction is an effective method for the production of aqueous extracts of propolis. The results show that depending on concentration, propolis extracts reduce the viability of keratinocytes. The phenolic compounds under observation penetrated the epidermis and dermis from designed formulations. The base of semi-solid formulation influences the efficacy of propolis preparations. The overall quantity of phenolic compounds that penetrated the skin was around 2 % from the ointment and 1.5 % from the cream.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Upregulation of p53 by tannic acid treatment suppresses the proliferation of human colorectal carcinoma<abstract><title style='display:none'>Abstract</title><p>The present study’s objective is to clarify the molecular mechanisms of tannic acid effects on the viability of human colorectal carcinoma (CRC). Tannic acid is stable for up to 48 h and is localized in both cytoplasm and nucleus. It dose-dependently inhibited the viability of CRC cell lines; SW-620 and HT-29 with <italic>IC</italic><sub>50</sub> values of 7.2 ± 0.8 and 37.6 ± 1.4 µmol L<sup>–1</sup>. Besides, metastatic, invasive, and colony formation properties of CRC cells were significantly inhibited following the tannic acid treatment (<italic>p</italic> &lt; 0.001). Tannic acid has been found to modulate enzyme, protein, and gene expressions of NQO1 in different levels and the upregulation of protein/gene expressions of p53 (<italic>p</italic> &lt; 0.001), which leads the cells to trigger apoptosis. In conclusion, the present <italic>in vitro</italic> study may supply a significant background for <italic>in vivo</italic> studies in which the molecular mechanisms of antioxidant and chemopreventive activities of tannic acid will completely clarify.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00Determination of penicillamine, tiopronin and glutathione in pharmaceutical formulations by kinetic spectrophotometry<abstract><title style='display:none'>Abstract</title><p>A novel and simple method for the determination of penicillamine (PEN), tiopronin (mercaptopropionyl glycine, MPG) and glutathione (GSH) in pharmaceutical formulations by kinetic spectrophotometry has been developed and validated. It is based on the redox reaction where the thiol compound (RSH) reduces Cu<sup>II</sup>-neocuproine complex to Cu<sup>I</sup>-neocuproine complex. The non-steady state signal of the formed Cu<sup>I</sup>- neocuproine complex is measured at 458 nm. The initial rate and fixed time (at 1 min) methods were validated. The calibration graph was linear in the concentration range from 8.0 × 10<sup>‒7</sup> to 8.0 × 10<sup>‒5</sup> mol L<sup>−1</sup> for the initial rate method and from 6.0 × 10<sup>‒7</sup> to 6.0 × 10<sup>−5</sup> mol L<sup>−1</sup> for the fixed time method, with the detection limits of 2.4 × 10<sup>−7</sup> and 1.4 × 10<sup>‒7</sup> mol L<sup>−1</sup>, resp. Levels of PEN, MPG and GSH in pharmaceutical formulations were successfully assayed by both methods. The advantages of the presented methods include sensitivity, short analysis time, ease of application and low cost.</p></abstract>ARTICLE2021-04-03T00:00:00.000+00:00The potential impact of gene therapy on health-related quality of life (HRQoL) domains in haemophilia<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Introduction</title><p>Haemophilia is an inherited bleeding disorder characterised by spontaneous bleeding, often leading to impaired health-related quality of life (HRQoL). Commonly used treatments include episodic and prophylactic treatment regimens. Gene therapies could soon become available, potentially creating a paradigm shift in patient management.</p></sec><sec><title style='display:none'>Aim</title><p>This paper proposes hypotheses about the potential impact of gene therapy on HRQoL domains in haemophilia, and how these impacts might differ compared with existing treatments.</p></sec><sec><title style='display:none'>Methods</title><p>An expert working group with 10 individuals experienced in haemophilia and HRQoL research was established to discuss potential impacts of gene therapy on HRQoL in general and for specific domains in haemophilia. As part of a one-day workshop, domains of three widely used patient-reported outcome (PRO) instruments were explored: the Haemo-QoL-A, the Patient Reported Outcomes, Burden and Experiences (PROBE), and the Haemophilia Activities List (HAL).</p></sec><sec><title style='display:none'>Results</title><p>The group expected a greater improvement in HRQoL from gene therapy compared with existing treatments for the following domains: physical/role functioning, worry, and consequences of bleeding (Haemo-QoL-A); haemophilia-related health and EQ-5D-5L (part of the PROBE); leg and arm function, and leisure activities (HAL). In contrast, the experts suggested that no change or potential deterioration might be observed for the emotional impact (HAL) and treatment concerns (Haemo-QoL-A) domains.</p></sec><sec><title style='display:none'>Conclusions</title><p>Current PRO instruments in haemophilia have limitations when applied in the context of gene therapy, and no single instrument fully captures the relevant HRQoL domains. However, the PROBE and Haemo-QoL-A were considered as the most suitable existing instruments. As haemophilia treatments evolve, further research should examine the potential effectiveness of existing PRO instruments as compared to the development of novel PRO measures.</p></sec></abstract>ARTICLE2021-05-30T00:00:00.000+00:00Web-based Application for the Population Pharmacokinetic Service (WAPPS)'s impact on dosage selection: a single paediatric centre experience<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title><p>Current treatment for severe haemophilia includes prophylactic factor replacement to prevent bleeding. Coagulation factor products have significant inter-patient variability in pharmacokinetic (PK) parameters. Optimal management requires tailoring prophylaxis to individual PK parameters. Web-based Application for the Population Pharmacokinetic Service (WAPPS) is a tool that estimates individual PK values using a population approach. Despite its growing use to help guide dosing selection, few studies have investigated its clinical impact.</p></sec><sec><title style='display:none'>Aim</title><p>To investigate any change in prophylaxis regimen and hours per week where factor level is under 1%, pre- and post-PK testing using WAPPS, for paediatric patients with severe haemophilia.</p></sec><sec><title style='display:none'>Methods</title><p>A retrospective chart review was conducted for all paediatric patients with severe haemophilia receiving care between April 2013 and July 2018 at McMaster Children's Hospital who have used WAPPS. Data extracted included: patient demographics, PK data generated by WAPPS, prophylaxis regimen pre- and post-PK testing, and reason for regimen change. The number of hours per week where factor level was under 1% pre- and post-PK testing was calculated using WAPPS.</p></sec><sec><title style='display:none'>Results</title><p>Thirty-one patients were included; 42% (n=13) changed their prophylaxis regimen after PK testing. After using PK data to personalise prophylaxis recommendations, there was a decrease in the number of hours per week where factor level is under 1% (from an average of 13.1 hours/week to 11.8 hours/week), though not statistically significant (p=0.16).</p></sec><sec><title style='display:none'>Conclusion</title><p>PK data generated by WAPPS has direct impact by informing changes to prophylaxis recommendations. This individualised approach promotes patient-centred care and patient engagement without increasing the time spent with factor levels below 1%. It also confirms and validates clinical practice.</p></sec></abstract>ARTICLE2021-07-13T00:00:00.000+00:00A descriptive study of United States bleeding disorders camps<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title><p>Disease-specific camps present one means of helping children overcome the challenges associated with chronic conditions and improving clinical and psychosocial outcomes. For more than 50 years, bleeding disorders camps (BDCs) in the United States (US) have been promoting independence, self-care, and leadership skills in children with bleeding disorders, all while fostering camaraderie in a secure and safe environment. However, little is known about how BDCs are organised, administered, funded, staffed, or how staff are compensated.</p></sec><sec><title style='display:none'>Aim</title><p>This article aims to describe the attributes of BDCs that service the US bleeding disorders community, and to compare and contrast these attributes to identify gaps in the BDC system and areas for improvement.</p></sec><sec><title style='display:none'>Methods</title><p>The National Hemophilia Foundation (NHF), in collaboration with several members of its Nursing Working Group and Physical Therapy Working Group, developed a survey that was distributed to BDC administrators (CAs) and health care providers (HCPs).</p></sec><sec><title style='display:none'>Results</title><p>A total of 101 HCPs and 20 CAs completed the survey. Findings indicated that BDCs are an informal extension of both the HTCs and NHF chapters, reaffirming that camps play a crucial role in the overall care of bleeding disorders. In general, diminishing financial resources threaten the existence of BDCs. Although there are BDC guidelines for formal staff training and specific interventions delivered to camp participants, adherence is variable. Other gaps included minimal self-infusion education follow-up with no documentation on effect or benefit of infusion education provided at camp.</p></sec><sec><title style='display:none'>Conclusion</title><p>Addressing the gaps identified by this survey and documenting resultant data supporting the value of BDCs will facilitate their continued sustainability in light of increasingly limited funding.</p></sec></abstract>ARTICLE2021-03-03T00:00:00.000+00:00Case report of nasal pseudotumor – a rare presentation in severe haemophilia A with high titre inhibitors<abstract><title style='display:none'>Abstract</title><p>Haemophilia patients with inhibitors suffer from increased morbidity and mortality due to the ineffectiveness of factor VIII replacement. Pseudotumors are rare but dangerous complications in these patients, and nasal pseudotumors are even rarer. Here, we present the case of a young child with severe haemophilia A with high titre inhibitors who developed a nasal pseudotumor. When immune tolerance therapy was not possible due to financial constraints, he was treated with FEIBA prophylaxis and rituximab. The pseudotumor was managed with surgical excision. We conclude that epistaxis in haemophiliacs can be due to an underlying nasal pseudotumor, and highlight the use of rituximab for the eradication of inhibitors.</p></abstract>ARTICLE2021-03-03T00:00:00.000+00:00The impact of heavy periods on women with a bleeding disorder<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title><p>Women with a bleeding disorder (WBD), including those diagnosed as a carrier, often have heavy periods associated with prolonged bleeding and pain. This survey sought to describe the impact of this substantial burden on daily living and the personal cost of managing heavy periods.</p></sec><sec><title style='display:none'>Methods</title><p>An online survey was promoted to women who identify as having a bleeding disorder via the social media of The Haemophilia Society in January and February 2020. The survey included 20 questions about personal data, symptoms and the practicalities of living with a bleeding disorder.</p></sec><sec><title style='display:none'>Results</title><p>A total of 181 responses were received, of which 151 were complete questionnaires. Of these, 58% of respondents were aged 18–45 and 136 identified as having a bleeding disorder, mostly haemophilia or von Willebrand disease. Thirteen (10%) had been diagnosed as a haemophilia carrier and a further four women were probable carriers. Prolonged or painful periods were reported by the majority of respondents; the median duration of bleeding was 7 days (range 2–42). Thirty-six per cent took time off work or study as a result and 42% reported a negative impact on social life. Eighteen women (13%) reported having to use a combination of sanitary protection products to manage their bleeding. Women diagnosed as a carrier reported morbidity comparable with that of women with a diagnosed bleeding disorder and reported greater use of combinations of sanitary protection.</p></sec><sec><title style='display:none'>Conclusion</title><p>WBD experience a high prevalence of heavy bleeding and prolonged, painful periods despite using appropriate symptomatic treatment. The impact of heavy periods on women diagnosed as a being a carrier is comparable with that experienced by women with a diagnosed bleeding disorder, but as they are not always clinically recognised they may lack access to care and support.</p></sec></abstract>ARTICLE2021-05-02T00:00:00.000+00:00Red Flag Study: An observational cross-sectional survey looking at bleeding in patients with a bleeding disorder who are lost to follow-up<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title><p>Regular follow-up visits and routine care is important for people with a mild bleeding disorder in terms of lowering their risk of complications from untreated bleeds and helping them maintain a healthy lifestyle. However, follow-up visits among this population can sometimes be missed for unclear reasons.</p></sec><sec><title style='display:none'>Aim</title><p>The present study aimed to question if lost-to-follow-up patients with a bleeding disorder experience unreported but important bleeding events that are not communicated to their haemophilia treatment centre (HTC) and if they could benefit from more frequent clinic visits.</p></sec><sec><title style='display:none'>Methods</title><p>A multicentre paper-based cross-sectional survey was sent to people diagnosed with an inherited blood disorder and lost to follow-up for two years or more. Those who met the eligibility criteria received the survey by mail and completed and returned it to their HTC between October 2015 and July 2016.</p></sec><sec><title style='display:none'>Results</title><p>Invitation packages were sent to 71 individuals; 14 questionnaires returned, with a survey response rate of 19.7%. Of the 14 returned surveys, only 11 participants were eligible who either responded completely or partially to the survey. Quality of life was reported as almost never or never a problem by all but one participant, who limited activities due to bleeding problems. Spontaneous nosebleeds were sometimes, often or always a problem for three participants; one female participant reported issues associated with heavy menstrual bleeding as often or almost always a problem.</p></sec><sec><title style='display:none'>Conclusion</title><p>We concluded that although the mean annual bleeding self-reported events were relatively low, they cannot be underestimated when keeping in mind the limitations and challenges of accessing data among this population. Our study highlighted the importance of educating this group of patients on their bleeding disorder and engaging them in their own care and health status, which may result in improving their health-related quality of life and overall health outcomes.</p></sec></abstract>ARTICLE2021-05-30T00:00:00.000+00:00Personalising haemophilia management with shared decision making<abstract><title style='display:none'>Abstract</title><p>The current standard of care for treating people with haemophilia (PWH) in the developed world is prophylaxis with regular infusions of clotting factor concentrates. Gene therapy is being investigated as a new treatment paradigm for haemophilia and if approved would potentially eliminate the need for chronic, burdensome infusions. In recent years, shared decision making (SDM) has become increasingly common in patient care settings. SDM is a stepwise process that relies on reciprocal information sharing between the practitioner and patient, resulting in health care decisions stemming from the informed preferences of both parties. SDM represents a departure from the traditional, paternalistic clinical model where the practitioner drives the treatment decision and the patient passively defers to this decision. As the potential introduction of gene therapy in haemophilia may transform the current standard of care, and impact disease management and goals in unique ways, both practitioners and PWH may find their knowledge tested when considering the appropriate use of a novel technology. Therefore, it is incumbent upon haemophilia practitioners to foster an open, trusting, and supportive relationship with their patients, while PWH and their caregivers must be knowledgeable and feel empowered to participate in the decision making process to achieve truly shared treatment decisions.</p></abstract>ARTICLE2021-06-18T00:00:00.000+00:00Dental extraction in congenital factor Vll deficiency with inhibitor – a case report<abstract><title style='display:none'>Abstract</title><sec><title style='display:none'>Background</title><p>Hereditary factor VII (FVII) deficiency is a rare bleeding disorder with autosomal recessive inheritance, and FVII deficiency with an inhibitor is extremely rare. There is sparse information in the literature on the management of tooth extraction in patients with FVII deficiency and an inhibitor.</p></sec><sec><title style='display:none'>Case description</title><p>We report the case of a five-year-old child with FVII deficiency and an inhibitor who underwent dental extraction. The child had had multiple bleeding episodes including intracranial haemorrhage and had a history of severe allergic reaction to the infusion of recombinant FVII. The tooth was extracted using lignocaine gel and the antifibrinolytic agent oral tranexamic acid.</p></sec><sec><title style='display:none'>Conclusion</title><p>The extraction of a deciduous tooth in a patient with FVII deficiency and an inhibitor was undertaken without bleeding complications. There are currently no guidelines regarding management of this type of case. Further studies and evidence are required so that management can be standardised.</p></sec></abstract>ARTICLE2021-05-30T00:00:00.000+00:00en-us-1