Emel OKULU, Ebru AZAPAĞASI, Ömer EREDEVE, Hasan AKDUMAN, Saadet ARSLAN, Tanıl KENDİRLİ, Oktay PERK, Gaffari TUNÇ, Begüm ATASAY, Fatma Tuba EMİNOĞLU

Continuous venovenous hemodiafiltration in the treatment of newborns with an inborn metabolic disease: a single center experience

Background/aim: Most inborn metabolic diseases are diagnosed during the neonatal period. The accumulation of toxic metabolitesmay cause acute metabolic crisis with long-term neurological dysfunction and death. Renal replacement therapy (RRT) modalities allowthe efficient removal of toxic metabolites. In this study, we reviewed our experience with continuous venovenous hemodiafiltration(CVVHDF) as RRT for newborns with an inborn metabolic disease.Materials and methods: Patients diagnosed with an inborn metabolic disease and who received CVVHDF treatment at our neonatalintensive care unit between January 2014 and December 2017 were included in this study. Their demographic and clinical data werecollected, and the efficacy and safety of CVVHDF was evaluated.Results: A total of nine continuous RRT (CRRT) sessions as CVVHDF were performed in eight newborns with a diagnosis of urea cycledefect (n = 5), maple syrup urine disease (n = 2), or methylmalonic acidemia (n = 1). The mean age at admission was 10 ± 8.6 days(range: 3–28 days). The mean plasma levels of ammonium were 1120 ± 512.6 mg/dL and 227.5 ± 141.6 mg/dL before and at the endof the treatment, respectively. Plasma levels of leucine were 2053.5 ± 1282 μmol/L and 473.5 ± 7.8 μmol/L before and at the end of thetreatment, respectively. The CVVHDF duration was 32.3 ± 11.1 h (median: 37 h; range: 16–44 h), and the mean length of hospitalizationwas 14.6 ± 12.9 days. The mean duration of CVVHDF was 32.3 ± 11.1 h (range: 16–44 h). Circuit clotting was the most commonobserved complication (37.5%) and the survival rate was 50%. Among surviving patients, two developed severe and two developed mildmental and motor retardation.Conclusion: CVVHDF is a CRRT modality that can be used to treat newborns with an inborn metabolic disease. Early diagnosis,commencement of specific medical therapy, diet, and extracorporeal support, if needed, are likely to result in improved short and longtermoutcomes.

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1. Arbeiter AK, Kranz B, Wingen AM, Wingen AM, Bonzel KE, et al. Continuous venovenous haemodialysis (CVVHD) and continuous peritoneal dialysis (CPD) in the acute management of 21 children with inborn errors of metabolism. Nephrology Dialysis Transplantation 2010; 25 (4): 1257-1265. doi: 10.1093/ ndt/gfp595.
2. Surtees RA, Matthews EE, Leonard JV. Neurologic outcome of propionic acidemia. Pediatric Neurology 1992; 8 (5): 333-337.
3. Msall M, Batshaw ML, Suss R, Brusilow SW, Mellits ED. Neurologic outcome in children with inborn errors of urea synthesis: Outcome of urea cycle enzymopathies. New England Journal of Medicine 1984; 310 (23): 1500-1505.
4. Hilliges C, Awiszus D, Wendel U. Intellectual performance of children with maple syrup urine disease. European Journal of Pediatrics 1993; 152 (2): 144-147.
5. Braun MC, Welch TR. Continuous venovenous hemodiafiltration in the treatment of acute hyperammonemia. American Journal of Nephrology 1998; 18 (6): 531-533.
6. Rutledge SL, Havens PL, Haymond MW, McLean RH, Kan JS, et al. Neonatal hemodialysis: Effective therapy for the encephalopathy of inborn errors of metabolism. Journal of Pediatrics 1990; 116 (1): 125-128.
7. Falk MC, Knight JF, Roy LP, Wilcken B, Schell DN, et al. Continuous venovenous haemofiltration in the acute treatment of inborn errors of metabolism. Pediatric Nephrology 1994; 8 (3): 330-333.
8. Hmiel SP, Martin RA, Landt M, Levy FH, Grange DK. Amino acid clearance during acute metabolic decompensation in maple syrup urine disease treated with continuous venovenous hemodialysis with filtration. Pediatric Critical Care Medicine 2004; 5 (3): 278-281.
9. Mathias RS, Kostiner D, Packman S. Hyperamonemia in urea cycle disorders: Role of the nephrologist. American Journal of Kidney Diseases 2001; 37 (5): 1069-1080.
10. Pela I, Seracini D, Donati MA, Lavoratti G, Pasquini E, et al. Peritoneal dialysis in neonates with inborn errors of metabolism: Is it really out of date? Pediatric Nephrology 2008; 23 (1): 163-168.
11. Donn SM, Swartz RD, Thoene JG. Comparison of exchange transfusion, peritoneal dialysis, and hemodialysis for the treatment of hyperammonemia in an anuric newborn infant. Journal of Pediatrics 1979; 95 (1): 67-70.
12. Sperl W, Geiger R, Maurer H, Guggenbichler JP. Continuous arteriovenous haemofiltration in hyperammonemia of newborn babies. Lancet 1990; 336 (8724): 1192-1193.
13. Rajpoot DK, Gargus JJ. Acute hemodialysis for hyperammonemia in small neonates. Pediatric Nephrology 2004; 19 (4): 390–395. doi: 10.1007/s00467-003-1389-5.
14. Ponikvar R, Kandus A, Urbancic A, Kornhauser AG, Primozic J, Ponikvar JB. Continuous renal replacement therapy and plasma exchange in newborns and infants. Artificial Organs 2002; 26 (2): 163-168.
15. MacDonald A, Dixon M, White F. Disorders of amino acid metabolism, organic acidaemias and urea cycle defects. In: Shaw V, Lawson M (editors). Clinical Paediatric Dietetics. 3rd ed. Oxford: Blackwell Publishing; 2007. pp. 309-375.
16. Häberle J. Clinical practice: the management of hyperammonemia. European Journal of Pediatrics 2011; 170 (1): 21-34. doi: 10.1007/s00431-010-1369-2.
17. Strazdins V, Watson AR, Harvey B. Renal replacement therapy for acute renal failure in children: European guidelines. Pediatric Nephrology 2004; 19 (2): 199-207. doi: 10.1007/ s00467-003-1342-7.
18. Warady B, Bunchman T. Dialysis therapy for children with acute renal failure: Survey results. Pediatric Nephrology 2000; 15 (1-2): 11-13.
19. Symons JM, Brophy PD, Gregory MJ, McAfee N, Somers MJ, et al. Continuous renal replacement therapy in children up to 10 kg. American Journal of Kidney Diseases 2003; 41 (5): 984-989.
20. Yetimakman AF, Kesici S, Tanyildiz M, Bayrakci US, Bayrakci B. A report of 7-year experience on pediatric continuous renal replacement therapy. Journal of Intensive Care Medicine 2017; 1:885066617724339. doi: 10.1177/0885066617724339.
21. Spinale JM, Laskin BL, Sondheimer N, Swartz SJ, Goldstein SL. High-dose continuous renal replacement therapy for neonatal hyperammonemia. Pediatric Nephrology 2013; 28 (6): 983- 986. doi: 10.1007/s00467-013-2441-8.
22. Chen CY, Chen YC, Fang JT, Huang CC. Continuous arteriovenous hemodiafiltration in the acute treatment of hyperammonaemia due to ornithine transcarbamylase deficiency. Renal Failure 2000; 22 (6): 823-836.
23. Ronco C, Parenzan L. Acute renal failure in infancy: Treatment by continuous renal replacement therapy. Intensive Care Medicine 1995; 21 (6): 490-499.
24. Schröder CH, Severijnen RS, Potting CM. Continuous arteriovenous hemofiltration (CAVH) in a premature newborn as treatment of overhydration and hyperkalemia due to sepsis. European Journal of Pediatric Surgery 1992; 2 (6): 368-369.
25. Picca S, Bartuli A, Dionisi-Vici C. Medical management and dialysis therapy for the infant with an inborn error of metabolism. Seminars in Nephrology 2008; 28 (5): 477-480. doi: 10.1016/j.semnephrol.2008.05.007.
26. Jouvet P, Poggi F, Rabier D, Michel JL, Hubert P, et al. Continuous venovenous haemodiafiltration in the acute phase of neonatal maple syrup urine disease. Journal of Inherited Metabolic Disease 1997; 20 (4): 463-472.
27. Picca S, Dionisi-Vici C, Abeni D, Pastore A, Rizzo C, et al. Extracorporeal dialysis in neonatal hyperammonemia: Modalities and prognostic indicators. Pediatric Nephrology 2001; 16 (11): 862-867.
28. Schaefer F, Straube E, Oh J, Mehls O, Mayatepek E. Dialysis in neoantes with inborn errors of metabolism. Nephrology Dialysis Transplantation 1999; 14 (4): 910-918.
29. Bishof NA, Welch TR, Sfrife CF, Ryckman FC. Continuous hemodiafiltration in children. Pediatrics 1990; 85 (5): 819-823.
30. Holmes CE, Huang JC, Cartelli C, Howard A, Rimmer J, et al. The clinical diagnosis of heparin-induced thrombocytopenia in patients receiving continuous renal replacement therapy. Journal of Thrombosis and Thrombolysis 2009; 27 (4): 406- 412. doi: 10.1007/s11239-008-0228-8.
31. Ferreira JA, Johson DW. The incidence of thrombocytopenia associated with continuous renal replacement therapy in critically ill patients. Renal Failure 2015; 37 (7): 1232-1236. doi: 10.3109/0886022X.2015.1057799.
32. Leonard JV, Morris AA. Diagnosis and early management of inborn errors of metabolism presenting around the time of birth. Acta Paediatrica 2006; 95 (1): 6-14.
33. Batshaw ML. Inborn errors of urea synthesis. Annals of Neurology 1994; 35 (2): 133-141.
34. McBryde KD, Kershaw DB, Bunchman TE, Maxvold NJ, Mottes TA, Kudelka TL, Brophy PD. Renal replacement therapy in the treatment of confirmed or suspected inborn errors of metabolism. Journal of Pediatrics 2006; 148 (6): 770- 778. doi: 10.1016/jpeds.2006.01.004.
35. Gündüz M, Ünal S, Okur I, Ayrancı Sucaklı I, Güzel F, Koç N. Neonates with inborn errors of metabolism: Spectrum and short-term outcomes at a tertiary care hospital. Turkish Journal of Pediatrics 2015; 57 (1): 45-52.
36. Kamate M, Chetal V, Kulgod V, Patil V, Christopher R. Profile of inborn errors of metabolism in a tertiary care centre PICU. Indian Journal of Pediatrics 2010; 77 (1): 57-60. Doi: 10.1007/ s12098-010-0008-2.