2017 X4 Pharmaceuticals Announces Initiation of a Phase II/III Clinical Study of X4P-001-LD in Patients with WHIM Syndrome, a Rare Genetic Primary Immunodeficiency Disease
WHIM is an abbreviation for the characteristic symptoms of the syndrome: Warts, Hypogammaglobulinemia, Infections and Myelokathexis. It is a rare primary immunodeficiency with an estimated incidence of 0.23 per million births and an unknown exact prevalence. Because patients are highly susceptible to infections, WHIM syndrome is associated with significant morbidity. While there is no approved treatment for WHIM syndrome, current therapy is limited to treatment of acute infections with antibiotics or prevention through the use of intravenous immune globulin therapy.
2017 Studies Shed Light on Effective Treatments for Kawasaki Disease Patients
Two studies, one that is completed and one that is just starting, are helping to shed light on the most effective treatments for Kawasaki disease (KD) patients.
One study that compared corticosteroids plus intravenous immune globulin (IVIG) therapy with IVIG therapy alone highlights the importance of timing to prevent coronary artery complication in treating high-risk KD patients. The meta-analysis reviewed 16 comparative studies involving 2,746 patients treated with either corticosteroids as initial therapy or as rescue therapy. Researchers found that the duration of illness before corticosteroids therapy was significantly shorter in the initial corticosteroids subset than in the rescue corticosteroids subset. And, the rate of coronary artery abnormalities was significantly lower in adjunctive corticosteroids therapy than in IVIG therapy alone. Subgroup analysis, including studies using corticosteroids plus IVIG as initial therapy, showed a more advantageous effect than IVIG alone regarding coronary artery abnormality prevention, whereas this benefit was not found in a subgroup of studies using corticosteroids as rescue therapy. Further analysis found that patients predicted at baseline to be at high risk of IVIG resistance seemed to obtain the greatest benefit from adjunctive corticosteroid therapy regarding coronary artery abnormality prevention.1
A new three-year study to be conducted by researchers at University of California San Diego School of Medicine, Rady Children’s Hospital-San Diego and Betty Irene Moore School of Nursing at University of California, Davis, funded with a $2 million grant from the Patient-Centered Outcomes Research Institute (PCORI), will look at the effectiveness of two treatment options for children with KD who are resistant to initial therapy. Standard treatment for KD is IVIG, but 10 percent to 20 percent of patients are resistant to the therapy, putting them at a higher risk for serious complications such as coronary artery damage and aneurysms. Currently, there are no guidelines for the best secondary treatment. Most patients receive either a second infusion of IVIG or an engineered antibody called infliximab that inactivates a molecule that promotes inflammation. The PCORI grant will support a study to compare the effectiveness of these two approaches for IVIG-resistant KD patients.
“After talking to more than 100 parents, clinicians and researchers, we learned that their top priority for research is to test the effectiveness of treatments to prevent heart damage in this fragile patient population,” said Jane Burns, MD, co-principal investigator of the study and professor of pediatrics at UC San Diego School of Medicine “Our findings will further Kawasaki disease research and give insight into how to approach patients who do not respond to initial treatment.”2
References 1. Chen, S, Dong, Y, Galindo Kiuchi, M, et al. Coronary Artery Complication in Kawasaki Disease and the Importance of Early Intervention: A Systematic Review and Meta-Analysis. JAMA Pediatrics, published online Oct. 17, 2016. Accessed at www.ncbi.nlm.nih.gov/pubmed/27749951. 2. UC San Diego Researcher to Study Most Effective Treatment for Kawasaki Disease. University of California, San Diego, press release Feb. 2, 2017. Accessed at www.eurekalert.org/pub_releases/2017-02/uoc--usd020217.php.