Jutte Van der Werff ten Bosch, BelgiumUZ BRussel Pediatrics
Presenter Of 4 Presentations
WELCOME FROM ESID 2019 MEETING CHAIR
SECONDARY IMMUNODEFICIENCY AFTER TREATMENT FOR CANCER
Exploiting the immune system in order to treat malignancy has been a longstanding field of interest. On the one hand, antibodies against a variety of more or less tumour specific antibodies have been developed, starting with anti CD20 or Rituximab. Nowadays, the field has moved forward, and a variety of different approaches is now available for the clinician, varying from biclonal antibodies, through CART cells, to small molecules targeted to proteins mutated in some malignancies but necessary for a functional immune system as well. As more and more patients survive, the interest to gain more insight in the behaviour of the immune system after therapy is growing. The lecture will talk you through the different therapies and the types of secondary immunodeficiency's that have been described. A second niche to be discussed is the growing insight in the evolution of the immune system after stemcell transplantation.
CHRONIC MUCOCUTANEOUS CANDIDIASIS IN A TODDLER WITH STAT1 GAIN-OF-FUNCTION MUTATION
Background and Aims
Chronic mucocutaneous candidiasis (CMC) is a heterogenous group of primary immunodeficiency diseases characterized by chronic and recurrent Candida infections primarily involving the nails, skin and mucous membranes. Impaired IL-17 T-cell immunity is known to be one of the mechanisms of this disease, as Th17 cells and their effector cytokines IL-17 and IL-22 have critical functions for candidial host defense via epithelial cells. Gain-of-function (GOF) mutations in the human signal transducer and activator of transcription 1 (STAT1) gene cause an impaired Th17 cell production and can cause CMC.
A two-year-old girl presented to our hospital at the age of 1,5 years with failure to thrive, recurrent bacterial respiratory tract infections, diarrhea and recurrent oral thrush, not responding well to local treatment. Oral mucosa swab repeatedly showed Candida albicans. She was born as the second child of healthy, non-consanguineous parents of Serbian origin. Initial immunological screening revealed a normal white blood cell count and normal serum immunoglobulines. Response to vaccination with Pneumovax 23 was normal. Further workup showed normal T cells with low Th17 cell counts (0,08% of CD4 T cells) and impaired T cell function. Subsequently a genetic workup was performed which showed a GOF mutation in the STAT1 gene. This mutation is reported to be pathogenic for CMC.
Recurrent oral Candidiasis after the age of one year is an alarm sign and indicative for immunodeficiency disorders such as CMC. It is important to perform an immunological workup including genetic testing for the involved genes in such cases.
PRIVIGEN, A NEW 10% LIQUID PREPARATION OF POLYVALENT HUMAN IGG FOR INTRAVENOUS ADMINISTRATION, CAUSES MORE HEADACHES IN PEDIATRIC PATIENTS THAN OTHER INTRAVENOUS IMMUNOGLOBULIN REPLACEMENT THERAPIES.
Background and Aims
Introduction : Patients suffering from diseases associated with poor immunoglobulins levels or function can be helped with an immunoglobulin replacement therapy. These are often life-long therapies. Headaches are the most common adverse events of intravenous immunoglobulins. Multiple manufacturers make IV immunoglobulins, each having a proper manufacturing proces. This leads to a difference in tolerability.
Purpose : To investigate if the new IVIG Privigen causes more headaches in pediatric patients than other IVIG such as Nanogam, Multigam and Kiovig.
Material and methods : This is a retrospective study : in the first part, the data of the children treated with IVIG since 2008 will be studied. We will look for the incidence of headaches in relation to the used product. The second part of the study is a survey data collection. It will aim to evaluate their headaches during/before/after the treatment. Other adverse events such as fatigue, nausea and fever will also be evaluated.
Results : 21.9% suffered from headaches with Privigen, 20.4% with non-Privigen IVIG. Only in 56.3% of the Privigen group and in 63.3% of the other group was there use of precautionary measures. There tend to be less headaches in patients getting higher dosis (p:0.045). Fatigue is the most common AE with 80.6% experiencing it. This was also the most invalidating AE, i.e. school absenteeism.
Conclusions : Privigen didn’t cause more headaches than the alternative IVIG products. On the other hand, fatigue is an important cause of morbidity in IVIG therapy. Precautionary measures should be used more.