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2nd Congress of the European Group – International Society for Apheresis

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03/22/2018 - Pirouette | 2:15pm - 3:45pm 
Apheresis and plasma exchange in children and adolescents

Chairs: S. Greber-Platzer (Vienna, Austria), C. Stefanutti (Rome, Italy)

Therapeutic apheresis in very low weight patients including newborns and children
*Claudia  Stefanutti1, Serafina Di Giacomo1, Claudia Morozzi1
1 Sapienza University of Rome, Molecular Medicine, ROMA, Italy
Abstract text :

The atherosclerotic process does not spare children, with the development of fatty streaks in the aorta already seen by 10 years of age and in the coronary arteries by 20 years of age. This highlights the importance of earlier and even more aggressive treatment and follow-up in HoFH and the need for additional lipid lowering therapies. This is reflected in the most recent statements on target levels for both homozygous and heterozygous FH. Also in children, Lipoprotein Apheresis (LA) can be very effective to lower LDL-c levels, and consequently CV risk. The EAS position paper on paediatric FH recommends to start LA as soon as technically possible in children with HoFH, and specifies that this may be as early as two years old in specialised centres. In HoFH, profound lowering of LDL by LA appears to improve coronary atherosclerosis and aortic valvular disease and increase longevity, particularly when treatment is initiated at an early age. Initiation of LA in very young, physically light children can be problematic, but is routinely achieved by skilled medical teams.

Familial hyperchylomicronemia  is an inherited disorder of lipoprotein metabolism characterized by elevated plasma chylomicrons and triglycerides, acute pancreatitis, cutaneous xanthomas, and hepatosplenomegaly. It is usually due to deficiency of lipoprotein lipase or its cofactor apolipoprotein C-II. Acute pancreatitis (AP) is a potentially life-threatening complication of severe hypertriglyceridaemia (sHTG). The standard therapeutic measures such as the use of lipid-lowering agents (fenofibrate, gemfibrozil, w-3 fatty acids), low molecular weight heparin, and insulin in diabetic patients in the above mentioned severe case often fail, at least as first approach in Emergency Department. Nonpharmacological approaches based upon the removal of TG with therapeutic plasma exchange, when indicated, can provide improvement of the clinical conditions in patients with sHTG, and profound, fast reduction of triglyceride-rich particles in plasma.

Psoriasiform hyperplasia of the epidermis and atopic dermatitis  is genetically determined, tremendous life-threatening disease in childhood due to elevated IgE in plasma. Skin is diffusely erythrodermic and uncontrollable itching reduces extremely the quality of life in affected children. Selective therapeutic apheresis which removes antibodies and circulating immune complex particles may help even in younger patients.

The use of therapeutic apheresis in very low weight patients is generally thought to have limitations, because of possible severe adverse reactions, potential risk related to the extracorporeal procedure, due to th elow weight of young patients. However, a careful therapeutic approach using appropriate precautions, and also introducing changes to the standard procedure, can minimise the risk without compromising the efficacy of therapeutic plasmapheresis.

Is the minimal residual disease (MRD) in HPC apheresis products in patients with neuroblastoma of importance for the prognosis. A retrospective study.
*Volker Witt1, Verena  Dastl1
1 St. Anna Kinderspital, , Vienna, Austria
Abstract text :

Background. Neuroblastoma is very rare in the total population but the most frequent extracranial solid tumor in childhood, arising in the embryonal remnants of the sympathetic nervous system. It´s clinical behavior is very diverse. Sometimes the tumor is curable treated with surgery only and others progress despite intensive chemotherapy, autologous stem cell transplantation and radiotherapy. Autologous stem cell rescue is a well-established therapy for children with high risk neuroblastomas but the mortality in children with metastatic disease, older than one year, is still high. The question arises whether residual neuroblastoma cells in autologous stem cell grafts increase the risk of relapse when being reinfused. Apheresis products, as well as bone marrow and peripheral blood, are analyzed to detect residual neuroblastoma cells, but the clinical significance of this minimal residual disease (MRD) and the impact on the outcome remain controversial.

Aim. In the following study the correlation between the MRD status in peripheral stem cell grafts (=apheresis product), bone marrow and peripheral blood will be investigated and the impact of MRD on outcome will be evaluated.

Design. Retrospective study.

Participants and methods. In total 228 apheresis products from 181 children with high-risk neuroblastoma were analyzed for minimal residual disease detection at the CCRI at the St. Anna Children"s Hospital from 2000 to 2017. Correlations between MRD status in apheresis products and bone marrow (n=100) or peripheral blood (n=39) were investigated. Overall survival depending on the MRD status was evaluated.

Results. Overall survival of patients observed in this study was 43,3% for female and 44,5% for male patients with significant differences within the age groups. Apheresis contamination was low (17/145 samples; 12%). Bone marrow contamination significantly correlated with the survival outcome (chi-square χ² test p=0,009) but apheresis contamination did not correlate with the outcome (Kaplan-Meier analysis p=0,780; χ² test p=0,584) although MRD status of apheresis correlated with the MRD status in BM (χ² test p=0,000). Peripheral blood contamination did neither correlate with apheresis contamination (χ² test p=0,767) nor with outcome (χ² test p=0,227).

Conclusion. Contamination of the apheresis product has no influence on the survival outcome but can be considered as an expression of high tumor burden or incomplete tumor eradication, whereas MRD status in the bone marrow can be considered as a strong predictor for outcome.

Pediatric apheresis – International survey
*Joseph Schwartz1
1 Columbia University, , New York, United States
Abstract text :

Pediatric apheresis has distinct characteristics compared to adult apheresis, and requires specialized knowledge and experience to perform safely, particularly in low-weight patients. However, there is wide variation in Pediatric apheresis practice worldwide. The American Society for Apheresis (ASFA) performed an electronic survey of >5,000 potential participants throughout the world to ascertain the scope and the current state of practice including type of procedures, age & weight of patients, and vascular access. The results of the survey will be discussed highlighting the scope and diversity of pediatric apheresis and identify areas where considerable variability in practice exists. Further exploration of these differences could establish best practices in pediatric apheresis through international research and collaboration.

Familial Hypercholesterolemia (FHC):Treatment by LDL aphaeresis: Lebanese experience
*Elie Moubarak1
1 National Lebanese LDL-apheresis center, , Beirut, Lebanon
Abstract text :

Objectives: In Lebanon, familial hypercholesterolemia shows an incidence about 25 times higher than in Europe, homozygous familial hypercholesterolemia has an estimated prevalence between 20 to 30 cases per one million inhabitants (Lebanese population: 4.7 million).
This high prevalence is due to intermarriage between family members (e.g. cousins) in isolated villages. There are currently 40-45 FHC patients treated to achieve better quality of life by reducing co-morbidity and mortality from cardio-vascular events.

Method: More than 14,400 sessions were performed between 2002 and 2017 (15 years of experience) for adults and children.
40 - 45 patients are regularly treated twice a month.
˂ 10 y.o. 2 patients, between 10-18 y.o 11 patients, between  19-35 y.o. 22 patients, between  36-60 y.o. 7 patients and only 2 patients are older than 60 y.o. The sex distribution is: 20 male and  24 female.
91% of the patients are homozygous with Low Density Lipoprotein LDL-cholesterol level > 600mgl/dl at date of arrival to the center and 9% of the patients are severe heterozygous.
All of the patients have severe hypercholesterolemia with LDL-C levels ranging from 420 mg to appx. 900 mg/dl despite conservative treatment (diet, resin, ezetimib, statins,Vit K2 & Q10 ,omega 3 6 9 , aspirin with or without clopidogrel). All of them have multiple xanthomas and ~ 2/3 0f  the patients have a lipid corneal arch .some of the patients lost family members due to Coronary Artery Disease (CAD) .
LDL apheresis was approved by the Food and Drug Administration (FDA) in 1996 for use in patients who despite diet and maximum tolerated drug therapy have  :LDL-C higher than 300 mg/dL in the absence of Coronary Heart Disease (CHD) and higher than 200 mg/dL when CHD is present. The procedure is usually performed in regional centers. A patient registry has been established to monitor prospectively clinical outcome and adverse effects.

Results: Acute LDL-C reductions in all 13 infants patients and 31 adults for a total of more than 14,400 sessions averaged 64-65 % sometimes up to 70 -80 % reduction of LDL

1. No deaths during session.
2. Overall physical improvement in the patients and some of them get married without any problem.
3. Disappearance of the angina and dyspnea.
4. Net regression of xanthomas and xanthelasmas.
5. Reduction of coronary events (no myocardial infarction has been reported in our patients).
6. Better exercise tolerance and Better Quality of Life.
7. All of the patients are improving and accepted the concept of a chronic apheresis treatment without any psychological problems and without fistula operations.
8. No serious adverse effects were observed;  
9. LDL-cholesterol reduction averaged up to 70 % (compared with 58% LDL-reduction in 2008).
10. The long term reduction of LDL-cholesterol was reduce the cardio-vascular events and reduces the mortality-morbidity of the patients.
11. Using Direct Adsorption of Lipoproteins (Dali) system is a well tolerated, safe, effective and a simple way of reducing LDL –C in patients not responding adequately to drug therapy.
12. No PCSK9 inh. and no register for FH in Lebanon.
13. LDL apheresis gives good long-term results both in adults, infants and in children.

Conclusion: LDL apheresis reduces cardiovascular events in hypercholesterolemic patients and may be an effective treatment for other vascular diseases including cholesterol embolic disease, focal segmental glomerular sclerosis, sudden hearing loss, and age-related macular degeneration.
Patients who are treated by DALI-apheresis  in our center over 15 years in a consistent and correct manner, the LDL remains low at home and never returns to the initial result (before LDL-apheresis).