Newsletter on Hemophilia Research: August-2018

Disruptive technology for the treatment of hemophilia

In patients with hemophilia A with inhibitors, a completely unique medical aid referred to as emicizumab decreases incidence of haemorrhage episodes by eighty seven %, a world team of hematologists has found. [1]

Value of prophylaxis vs on‐demand treatment: Application of a value framework in hemophilia

Introduction: Therapeutic advances over the past thirty years have light-emitting diode to longer anticipation and improved quality of life (QOL) for persons with bleeder’s disease. Access to innovative medical care could also be compromised if treatment selections area unit driven exclusively by price. New ways area unit required to assess true therapeutic values, beside money price, as physicians, policymakers, payers and makers work along to enhance patient care.

Aim: To provide an evidence‐based assessment of the value of prophylaxis vs on‐demand therapy for hemophilia, based on a widely recognized three‐tiered value framework approach for assessing a range of therapeutic interventions. [2]

Clinical behavior and complications of hemophilia in the pediatric population

Introduction: sex-linked disorder is Associate in Nursing congenital disease connected to sex, caused by the attenuated coagulator activity of antihemophilic globulin (hemophilia A), factor IX (hemophilia B) and plasma thromboplastin antecedent (hemophilia C). Its treatment represents a high economic value for the country and it’s related to nice frequency to serious disabilities of the afflicted patient, negatively impacting its bio-psychosocial development, additionally as Associate in Nursing adequate acquainted practicality.

Objective: to characterize sex-linked disorder clinically and epidemiologically within the medical specialty population of Pinar Del Rio province, within the amount from January 1985 to June 2016.

Method: Associate in Nursing experimental, descriptive and cross-sectional study was conducted in medical specialty patients diagnosed with sex-linked disorder. the information were extracted from the clinical files. The diagnosing was established by coagulogram, indefinite quantity of plasma factors VIII, IX and XI and indefinite quantity of inhibitors. Descriptive statistics was used. [3]

Rate and appropriateness of polypharmacy in older patients with hemophilia compared with age‐matched controls

Background: In older individuals, multiple chronic ailments result in the intake of multiple medications (polypharmacy) that carry variety of negative consequences (adverse events, prescription and intake errors, poor adherence, higher mortality). Because ageing patients with haemophilia (PWHs) may be particularly at risk due to their pre‐existing multiple comorbidities (arthropathy, liver disease), we have a tendency to selected to analyse the pattern of chronic drug intake in a very cohort of PWHs aged sixty years or additional.

Patients and methods: S + PHERA may be a multicentre empiric study, with the broad goal to evaluate prospectively the health status and medication intake in 102 older patients with severe haemophilia A or B compared with 204 age‐ and residence‐matched controls chosen randomly from the same general practices of PWHs. [4]

Higher Ratio of Arterial vs Venous Thrombosis in Hemophilia A as Compared with Von Willebrand Disease

Objectives: to analyze the magnitude relation between blood vessel and phlebothrombosis in patients with classical haemophilia or B as compared therewith of patients with Willebrand illness.

Methods: associate degreealysis of a time unlimited survey of the literature beside an analysis of non-public records. Thrombotic events had to be proved  by objective strategies. noninheritable  forms were excluded. 222 patients were found to fulfill the inclusion criteria, 131 patients with classical haemophilia or B and ninety one patients with Willebrand illness. [5]

Reference

[1] Disruptive technology for the treatment of hemophilia

Date:July 10, 2017Source:Children’s Hospital Los Angeles (web link)

[2] Value of prophylaxis vs on‐demand treatment: Application of a value framework in hemophilia

Nugent D, O’Mahony B, Dolan G, International Haemophilia Access Strategy Council. Value of prophylaxis vs on‐demand treatment: Application of a value framework in hemophilia. Haemophilia. 2018 Aug 8. (web link)

[3] Clinical behavior and complications of hemophilia in the pediatric population

Hernández Gonzalez JL, Campo Díaz M, Valdés Sojo C, Borrego Cordero G, Cabrera Morales C. Clinical behavior and complications of hemophilia in the pediatric population. Revista de Ciencias Médicas de Pinar del Río. 2018;22(2):226-33. (web link)

[4] Rate and appropriateness of polypharmacy in older patients with hemophilia compared with age‐matched controls

Mannucci PM, Nobili A, Marchesini E, Oliovecchio E, Cortesi L, Coppola A, Santagostino E, Radossi P, Castaman G, Valdrè L, Santoro C. Rate and appropriateness of polypharmacy in older patients with hemophilia compared with age‐matched controls. Haemophilia. 2018 Aug 16. (web link)

[5] Higher Ratio of Arterial vs Venous Thrombosis in Hemophilia A as Compared with Von Willebrand Disease

A. Girolami1*, E. Cosi1, L. Sambado1, V. Daidoneand F. Fabris1

1Department of Medicine, University of Padua Medical School, Padua, Italy. (web link)