Inhibitor in Congenital Factor VII Deficiency; a Rare but Serious Therapeutic Challenge-A Systematic Literature Review
Journal of clinical medicine. 2021;10(2)
BACKGROUND Congenital factor (F) VII deficiency is a rare coagulation factor deficiency with an estimated incidence of 1 per 500,000 individuals. Patients with severe FVII deficiency present a broad range of clinical presentations. Alloimmunization against exogenous FVII, as the main challenge of replacement therapy, is an extremely rare phenomenon that is accompanied by a high rate of life-threatening bleeding, that renders replacement therapy less effective. Due to the importance of the issue, we performed a systematic literature review in order to assess incidence, molecular basis, clinical presentations, and therapeutic challenge and management of inhibitor in congenital FVII deficiency. Strategy of search: This systematic review was performed in accordance with PRISMA guidelines. We performed an English-language literature review in the PubMed, EMBASE, Scopus, and Google Scholar databases, using the following keywords: "factor VII inhibitor", "factor VII inhibitors", "FVII inhibitors", "congenital FVII deficiency", "recombinant factor VII", "anti rFVIIa", "replacement therapy", and "alloantibody". RESULTS Out of 380 patients in the 13 studies, 27 had inhibitor against FVII; 18 were male, 7 were female, while the sex of 2 was not stated. The majority (92%) developed a high-titer inhibitor (Bethesda Unit > 5). All patients had severe FVII deficiency (FVII:C < 10%), and the majority received recombinant FVII prior to inhibitor development (N: 24, 89%). Among ten patients with a detected mutation, three subjects had a common non-sense (30%), and two had a deletion (20%). CONCLUSIONS Inhibitor development is a relatively rare phenomenon seen only in severe FVII deficiency, where it is associated with severe and life-threatening presentations, treatment challenge, and economic burden on the patients and their families.
Mobilization of the outer cavernous membrane decreases bleeding and improves resection in spheno-clinoidal meningiomas without cavernous sinus extension: A randomized controlled trial
Neurology India. 2018;66((2)):407-415.
Objective: The aim of this study was to determine whether adding mobilization of the outer cavernous sinus membrane as a part of the approach, in large spheno-clinoidal meningiomas without cavernous sinus extension, would reduce bleeding and increase the extent of resection. Methods:: This prospective randomized controlled trial was held between February 2016 and April 2017 at Cairo University Hospitals. The study recruited 94 patients with spheno-clinoidal meningiomas without cavernous sinus involvement. Patients were randomly assigned (by a computer based randomization system) into two groups; the treatment group, in which the patients received mobilization of the outer layer of the lateral wall of the cavernous sinus, prior to opening of the dura; and, the control group, in which the patients were operated by a direct opening of the dura without cavernous sinus dissection. The primary outcome of this study was the difference in the amount of blood lost during surgery between both groups of patients. The secondary outcome variables were the estimated blood loss (EBL) calculated according to Mercurelli's formula, the extent of tumor resection and the amount of blood transfused. Results:: The amount of blood loss and estimated blood loss (EBL) were significantly less in the "with mobilization group" with the P value being 0.00 and 0.013, respectively. Additionally, the amount of residual tumor was compared between both the groups and it showed that the group of patients who have received mobilization of the outer cavernous sinus membrane had a higher rate of radical resection as expressed by a lower volume of residual tumor (P value 0.005). Conclusion:: In large spheno-clinoidal meningiomas without cavernous sinus involvement, routine mobilization of the outer cavernous sinus membrane reduces bleeding. This helps in a better visualization of cranial nerves in a relatively avascular field as it enables the performance of neurovascular dissection in an earlier phase of surgery. It also enables a more radical resection.
Efficacy and safety of oral iron chelating agent deferiprone in beta-thalassemia and hemoglobin E-beta thalassemia
Indian Pediatrics. 1995;32((8):):855-61.
OBJECTIVES To assess efficacy and safety of oral iron chelating agent deferiprone (DFP) in patients with beta thalassemia and hemoglobin E-beta thalassemia. DESIGN Non-randomized study. SETTING Hematology Out-Patient Department. SUBJECTS Forty-one patients of beta thalassemia and hemoglobin E-beta thalassemia. INTERVENTIONS DFP was given to 20 patients, 10 patients of beta thalassemia and 10 with hemoglobin E-beta thalassemia; the rest were taken as controls. RESULTS A significant fall in serum ferritin was observed in the study group along with rise in urinary iron excretion (p < 0.05). Adverse effects of DFP were nausea and vomiting (30%), significant arthropathy requiring stopping of the drug (30%), and reversible neutropenia in one patient. All these complications could be managed easily with medical supervision and no death or permanent disability was seen. CONCLUSIONS DFP is an effective and fairly well tolerated oral iron chelating agent. The side effects that occur can be tackled easily if monitored properly.