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Background and Objectives: Plasma‐derived medicinal products (PDMPs) are essential to treat many chronic conditions such as haemophilia and primary immunodeficiency. Patients living in low middle‐income and low‐income countries (LMICs and LICs, respectively) have limited access to PDMPs. The aim of this article is to explore the challenges of accessing PDMPs in LMICs and LICs. Materials and Methods: A review of the literature and reports on blood safety, plasma production and its utilization to produce PDMPs in LMICs and LICs was carried out. Results: There is huge wastage of recovered plasma in LMICs and LICs as a result of a lack of good manufacturing practice (GMP) in the production of plasma for fractionation. Together with the high cost of imported PDMP procurement, patients have limited access to such products. Conclusion: There is a need to improve the situation by using domestically sourced plasma through the initiation of local plasma programmes through a stepwise approach to improve access to PDMPs in LMICs and LICs. [ABSTRACT FROM AUTHOR]

Shockingly, the global identification rate for haemophilia patients stands at only 31%, with the lowest rates observed in regions like Africa (8%) and Southeast Asia (17%),[3] and the identification rates for other bleeding disorders, including VWD, are even significantly lower. Dear Editor, Prompt and accurate diagnosis is crucial in effectively managing haemophilia and Von Willebrand Disease (VWD).[[1]] In haemophilia, delayed diagnosis or inadequate management can result in serious consequences such as life-threatening bleeding events leading to joint damage and disability, as well as the consequences of iron deficiency. A foundation to better care: Target product profile for haemophilia and Von Willebrand disease point-of-care diagnostics in low-resource settings In conclusion, the development of a TPP for POC devices designed specifically for haemophilia and VWD diagnosis in low-resource settings is a critical step towards addressing the challenges faced in diagnosing inherited bleeding disorders. [Extracted from the article]

Introduction: Prophylaxis has become standard of care for persons with severe phenotype haemophilia (PWsH). However, 'standard prophylaxis' with either factor or non‐factor therapies (emicizumab) is prohibitively expensive for much of the world. We sought to evaluate whether haemophilia care can be provided at a lower cost yet achieve good results using Lower dose/Lower frequency prophylaxis (LDP) and with increasing use of antifibrinolytics (Tranexamic acid and Epsilon amino caproic acid). Methods: We identified 12 studies that collectively included 335 PWsH using LDP. Additionally, we undertook a literature search regarding the benefits of antifibrinolytics in haemophilia care. Results: Identified studies show that LDP is far superior to no prophylaxis (On demand [OD] therapy) resulting in significant patient benefits. Patients on LDP showed (in comparison to patients OD) on average: 72% less total bleeds; 75% less joint bleeds; 91% less days lost from school; 77% less hospital admission days; and improved quality of life measures. These benefits come at similar or only slightly higher (< 2‐fold greater) costs than OD therapy. Antifibrinolytics are effective adjunctive agents in managing bleeds (oral, nasal, intracranial, possibly other) and providing haemostasis for surgeries (particularly oral surgeries). Antifibrinolytics can substitute for more expensive factor concentrates or can reduce the use of such concentrates. There is evidence to show that antifibrinolytics may be used in conjunction with factor concentrates/emicizumab for more effective/less costly prophylaxis. Conclusions: The use of LDP along with appropriate and increased use of antifibrinolytics offers less resourced countries good options for managing patients with haemophilia. [ABSTRACT FROM AUTHOR]

Despite the faster inhibitor eradication with high-dose ITI, a low-dose regimen has proven effective as well.6 We describe the experience of a low-dose regimen ITI with plasma-derived CFC in a real-world setting. Immune Tolerance Induction (ITI) remains the treatment of choice to achieve the latter goal.5 There are several ITI regimens, all of which involve frequent infusions of CFC to induce immune tolerance. Low-dose ITI is recommended in good-risk patients.5However, we opted for a low-dose regimen for all our patients, mainly due to cost effectiveness. Besides, evidence has shown that the low-dose ITI regimen is as effective as the high-dosing regimen.6 The I-ITI study showed that low-dose ITI takes longer to achieve immune tolerance and is associated with more bleedings.6 Regardless of the regimen, ITI should last no longer than 36 months.10 However, it was prolonged beyond 36 months in two patients. [Extracted from the article]

Heavy menstrual bleeding (HMB) is the commonest bleeding symptom among women with inherited bleeding disorders (IBD). Since HMB starts at the very onset of menarche and continues throughout the reproductive life, the health related quality of life of these women is affected and they are at an increased risk of developing iron‐deficiency anemia. Because of the entrenched stigma and taboos, women and girls are often reluctant to discuss the problem of HMB within their families and do not seek medical advice. Increased awareness and multidisciplinary management approach for the management of these women are essential in ensuring an optimal outcome. It is important to take a careful history and undertake a thorough gynecological assessment to exclude other underlying/concomitant causes of HMB. Iron supplementation is essential. Strategies for decreasing menstrual blood flow are similar to those used for HMB in general with the addition of desmopressin and replacement therapy and the exclusion of non‐steroidal anti‐inflammatory drugs. Tranexamic acid and/or hormonal intervention are usually recommended as first‐line therapy. Treatment choice should be individualized taking into account whether the woman wishes to preserve her fertility, if she requires contraception, the type of IBD, the severity of bleeding, and her social and religious background as well as acceptability and availability of the treatment options. [ABSTRACT FROM AUTHOR]

The aims of this study are to determine seroprevalence of Hepatitis E virus (HEV) in Tunisian blood donors and to evaluate its risk of parenteral transmission. Sera collected from 426 blood donors were tested for HEV IgG by indirect ELISA. Individuals were recruited from two national transfusion centers, in the North and the South of the country. Seroprevalence of HEV IgG was then compared with two other groups with increased risk of exposure to parenterally transmitted agents: 80 hemophiliac and 286 hemodialysis patients. Among blood donors, the seroprevalence was estimated to be 4.5%. It was significantly higher in the hemophiliac and hemodialysis groups with 7.5% and 10.2%, respectively, ( P = 0.002). No significant correlation was observed for this IgG 1 seroprevalence between age and sex among three studied groups. These results suggest that HEV has a high risk of parenteral transmission and confirm that the low endemicity of hepatitis E in Tunisia was observed. J. Med. Virol. 87:441-445, 2015. © 2014 Wiley Periodicals, Inc. [ABSTRACT FROM AUTHOR]

Prophylaxis is the gold standard treatment for haemophilia but requires more amount of clotting factor concentrates, than on demand therapy. Low dose prophylaxis is an alternative for countries with limited resources. There are data of evidence showing the superiority of low‐dose prophylaxis than episodic treatment. Studies from China, India, Tunisia, Thailand and Indonesia reported experiences with low dose prophylaxis using outcome assessment. These studies have shown the effectiveness of various protocols regimen with once, twice or thrice injection of 10‐15 UI Kg‐1 per injection. These protocols allow reduction of joint bleeds and at least delay of joint damages. There is not enough long‐term data nowadays, but low dose prophylaxis is certainly better than on demand therapy and should be considered as a first step of prophylaxis in some countries but not the final goal. [ABSTRACT FROM AUTHOR]