Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region.

Background: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript was to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region.

Transcranial Doppler and brain MRI in children with sickle cell disease and high hemoglobin F levels.

Background/objective: While overt stroke and silent brain infarcts (SBI) are uncommon among Kuwaiti patients with sickle cell disease (SCD), there have been no previous transcranial Doppler (TCD) studies in this population. The main objective of this study is to determine TCD velocities in a group of Kuwaiti children with SCD and correlate same with brain magnetic resonance imaging (MRI) and angiography (MRA) findings.

Materials And Methods: Forty-three steady-state, pediatric patients with SCD aged 10.

Infectious etiologies of transient neutropenia in previously healthy children.

Background: Healthy children presenting with neutropenia are often hospitalized and treated empirically with antibiotics without an evidence of infection. The objective of this study was to investigate the infectious causes of isolated transient neutropenia in otherwise previously healthy children.

Method: A 2-year prospective study was conducted at a tertiary hospital in Kuwait.

Does elevated hemoglobin F modulate the phenotype in Hb SD-Los Angeles?

Hemoglobin (Hb) SD-Los Angeles compound heterozygotes usually have a severe clinical course although the effect of an elevated Hb F on the clinical phenotype has not been reported previously. We describe 5 Kuwaiti children with Hb SD associated with Hb F levels >20%. They all presented with sickling-related symptoms by < or =2 years of age and have been followed for 3-15 years.

Undetected factor VIII in a patient with type 3 von Willebrands disease mistaken as severe haemophilia A.

von Willebrand disease (VWD) type 3 is a rare disorder characterized by absent or <0.1 UmL(-1) of ristocetin cofactor (VWF:RCo), and a very low level of factor VIII (FVIII:C). A total absence of FVIII:C has never been reported in type 3 VWD.