Efficacy of emicizumab in von Willebrand disease (VWD) patients with and without alloantibodies to von Willebrand factor (VWF): Report of two cases and review of literature.

INTRODUCTION: von Willebrand disease (VWD) is the common bleeding disorder with a clinically relevant bleeding prevalence of 1:10,000. von Willebrand disease patients lack both von Willebrand factor (VWF) and factor VIII (FVIII), which are critical for normal haemostasis. The conventional treatment for VWD includes desmopressin and replacement therapy with plasma derived FVIII with VWF concentrates or recombinant VWF. Development of alloantibodies is a rare occurrence, there is a paucity in the literature of treatment modalities in these patients. Not many reports are available in literature on the efficacy of emicizumab in VWD patients with or without alloantibodies to VWF. AIM: To do systematic review of literature on emicizumab in VWD and report our experience of emicizumab in two patients of VWD METHODS: We used electronic search engines till May 2021 in 'Google scholar' and 'PubMed', to collect the case reports or case series on use of emicizumab for management of VWD. Two of our severe VWD patients were successfully treated with emicizumab. A systematic review was performed and the results discussed. RESULTS: The electronic search revealed six case reports using emicizumab for treatment of VWD. Two were in vitro studies and four in patients with VWD type 3 disease. In vitro studies and in VWD patients on emicizumab, showed improvement in thrombin generation and fibrin formation. Among four patients, three had alloantibodies to VWD and one was negative. All these patients were treated with emicizumab for 6-12 m. After starting emicizumab, none of them had spontaneous bleeding requiring treatment. During treatment with emicizumab, one patient had trauma-associated soft tissue hematoma, which was treated with rFVIIa and another patient had bleeding following dental exfoliation treated with Humate P. We treated two of our VWD patients one with and one without inhibitors with emicizumab after failure of other therapies. Both the patients showed marked improvement and continued to remain well and free of bleeding episodes. None of the patients had any thrombosis or thrombotic microangiopathy (TMA) during treatment with emicizumab. CONCLUSION: In conclusion, this review supports the safety and efficacy of emicizumab in type 3 VWD patients with or without alloantibodies. Further large studies are required to confirm the safety and efficacy of emicizumab in VWD.

Dental Management of Factor XIII Deficiency Patients: A Case Series.

AIM: To create awareness about rare clotting disorders in children and to highlight the different dental treatment approaches that can be used while planning the management in such cases. BACKGROUND: A prerequisite for successful wound healing is achieving good hemostasis by effective vascular spasm, platelet plug formation, and finally blood coagulation. In the general population, postoperative bleeding after dental treatment is self-limiting. However, a certain segment of the population does suffer from inherited bleeding and clotting disorders, where in standard invasive dental procedures can trigger bleeding episodes, which could be life-threatening in absence of the requisite precautionary measures being followed. CASE DESCRIPTION: One such condition is congenital factor XIII deficiency, a rare autosomal recessive disease usually associated with early onset of serious or life-threatening bleeding episodes, such as, intracranial hemorrhage or umbilical cord bleeding. This case series details the complete dental management of three children suffering from factor XIII deficiency. CONCLUSION: Factor XIII is a rare coagulation cascade, and by this case series, complete dental treatment varying from noninvasive to be kept under observation to invasive dental extraction and fracture reduction was carried out with the hematologists consultations. CLINICAL SIGNIFICANCE: This case series creates awareness about this rare condition and the need for a multidisciplinary approach involving dentists and hematologists in the effective management of such patients. HOW TO CITE THIS ARTICLE: Pai NG, Mehta LK, Padhye NM, et al. Dental Management of Factor XIII Deficiency Patients: A Case Series. Int J Clin Pediatr Dent 2020;XX(X):1-4.