References
1. Schwab M, Westermann F, Hero B, Berthold F. Neuroblastoma: biology
and molecular and chromosomal pathology. Lancet Oncol . Aug
2003;4(8):472-80. doi:10.1016/s1470-2045(03)01166-5
2. Jokiranta TS. HUS and atypical HUS. Blood . May 25
2017;129(21):2847-2856. doi:10.1182/blood-2016-11-709865
3. Elfeky R, Lucchini G, Lum S-H, et al. New insights into risk factors
for transplant-associated thrombotic microangiopathy in pediatric HSCT.Blood Advances . 2020;4(11):2418-2429.
doi:10.1182/bloodadvances.2019001315
4. Cohn SL, Pearson AD, London WB, et al. The International
Neuroblastoma Risk Group (INRG) classification system: an INRG Task
Force report. J Clin Oncol . Jan 10 2009;27(2):289-97.
doi:10.1200/jco.2008.16.6785
5. Bu F, Borsa NG, Jones MB, et al. High-Throughput Genetic Testing for
Thrombotic Microangiopathies and C3 Glomerulopathies. J Am Soc
Nephrol . Apr 2016;27(4):1245-53. doi:10.1681/asn.2015040385
6. The PyMOL Molecular Graphics System. Version 2.0.
7. Park JR, Kreissman SG, London WB, et al. Effect of Tandem Autologous
Stem Cell Transplant vs Single Transplant on Event-Free Survival in
Patients With High-Risk Neuroblastoma: A Randomized Clinical Trial.Jama . Aug 27 2019;322(8):746-755. doi:10.1001/jama.2019.11642
8. Jodele S, Laskin BL, Dandoy CE, et al. A new paradigm: diagnosis and
management of HSCT-associated thrombotic microangiopathy as multi-system
endothelial injury. Blood reviews . 2015;29(3):191-204.
9. Harding M, Deyell RJ, Blydt-Hansen T. Catecholamines in
neuroblastoma: Driver of hypertension, or solely a marker of disease?Cancer Rep (Hoboken) . Aug 2022;5(8):e1569. doi:10.1002/cnr2.1569
10. Nakagawara A, Li Y, Izumi H, Muramori K, Inada H, Nishi M.
Neuroblastoma. Jpn J Clin Oncol . Mar 1 2018;48(3):214-241.
doi:10.1093/jjco/hyx176
11. Zheng XL. ADAMTS13 and von Willebrand factor in thrombotic
thrombocytopenic purpura. Annu Rev Med . 2015;66:211-25.
doi:10.1146/annurev-med-061813-013241
12. Karpman D, Manea M, Vaziri-Sani F, Ståhl AL, Kristoffersson AC.
Platelet activation in hemolytic uremic syndrome. Semin Thromb
Hemost . Mar 2006;32(2):128-45. doi:10.1055/s-2006-939769
13. Higham CS, Collins G, Shimano KA, et al. Transplant-associated
thrombotic microangiopathy in pediatric patients: pre-HSCT risk
stratification and prophylaxis. Blood Adv . Apr 27
2021;5(8):2106-2114. doi:10.1182/bloodadvances.2020003988
14. Palma LMP, Sridharan M, Sethi S. Complement in Secondary Thrombotic
Microangiopathy. Kidney Int Rep . Jan 2021;6(1):11-23.
doi:10.1016/j.ekir.2020.10.009
15. Zipfel PF, Wiech T, Stea ED, Skerka C. CFHR Gene Variations Provide
Insights in the Pathogenesis of the Kidney Diseases Atypical Hemolytic
Uremic Syndrome and C3 Glomerulopathy. J Am Soc Nephrol . Feb
2020;31(2):241-256. doi:10.1681/asn.2019050515
16. Boon CJF, van de Kar NC, Klevering BJ, et al. The spectrum of
phenotypes caused by variants in the CFH gene. Molecular
Immunology . 2009/05/01/ 2009;46(8):1573-1594.
doi:https://doi.org/10.1016/j.molimm.2009.02.013
17. Noris M, Bresin E, Mele C, Remuzzi G. Genetic Atypical
Hemolytic-Uremic Syndrome. In: Adam MP, Everman DB, Mirzaa GM, et al,
eds. GeneReviews(®) . University of Washington, Seattle.
18. Noris M, Caprioli J, Bresin E, et al. Relative role of genetic
complement abnormalities in sporadic and familial aHUS and their impact
on clinical phenotype. Clin J Am Soc Nephrol . Oct
2010;5(10):1844-59. doi:10.2215/cjn.02210310
19. Raina R, Vijayvargiya N, Khooblall A, et al. Pediatric Atypical
Hemolytic Uremic Syndrome Advances. Cells . Dec 18
2021;10(12)doi:10.3390/cells10123580
20. Tolbert VP, Dvorak CC, Golden C, et al. Risk Factors for
Transplant-Associated Thrombotic Microangiopathy after Autologous
Hematopoietic Cell Transplant in High-Risk Neuroblastoma. Biol
Blood Marrow Transplant . Oct 2019;25(10):2031-2039.
doi:10.1016/j.bbmt.2019.06.006
21. Jodele S, Dandoy CE, Myers K, et al. High-dose
Carboplatin/Etoposide/Melphalan increases risk of thrombotic
microangiopathy and organ injury after autologous stem cell
transplantation in patients with neuroblastoma. Bone Marrow
Transplant . Oct 2018;53(10):1311-1318. doi:10.1038/s41409-018-0159-8
22. Legendre CM, Licht C, Muus P, et al. Terminal complement inhibitor
eculizumab in atypical hemolytic-uremic syndrome. N Engl J Med .
Jun 6 2013;368(23):2169-81. doi:10.1056/NEJMoa1208981
23. Raina R, Krishnappa V, Blaha T, et al. Atypical Hemolytic-Uremic
Syndrome: An Update on Pathophysiology, Diagnosis, and Treatment.Ther Apher Dial . Feb 2019;23(1):4-21. doi:10.1111/1744-9987.12763
24. Dvorak CC, Higham C, Shimano KA. Transplant-associated thrombotic
microangiopathy in pediatric hematopoietic cell transplant recipients: a
practical approach to diagnosis and management. Frontiers in
Pediatrics . 2019;7:133.
25. Fakhouri F, Fila M, Hummel A, et al. Eculizumab discontinuation in
children and adults with atypical hemolytic-uremic syndrome: a
prospective multicenter study. Blood . May 6
2021;137(18):2438-2449. doi:10.1182/blood.2020009280
26. Syed YY. Ravulizumab: A Review in Atypical Haemolytic Uraemic
Syndrome. Drugs . Apr 2021;81(5):587-594.
doi:10.1007/s40265-021-01481-6
27. Tanaka K, Adams B, Aris AM, et al. The long-acting C5 inhibitor,
ravulizumab, is efficacious and safe in pediatric patients with atypical
hemolytic uremic syndrome previously treated with eculizumab.Pediatr Nephrol . Apr 2021;36(4):889-898.
doi:10.1007/s00467-020-04774-2
28. Nakamae H, Yamane T, Hasegawa T, et al. Risk factor analysis for
thrombotic microangiopathy after reduced-intensity or myeloablative
allogeneic hematopoietic stem cell transplantation. Am J Hematol .
Jul 2006;81(7):525-31. doi:10.1002/ajh.20648
29. Jodele S, Dandoy CE, Lane A, et al. Complement blockade for TA-TMA:
lessons learned from a large pediatric cohort treated with eculizumab.Blood . Mar 26 2020;135(13):1049-1057.
doi:10.1182/blood.2019004218
30. Dandoy CE, Rotz S, Alonso PB, et al. A pragmatic multi-institutional
approach to understanding transplant-associated thrombotic
microangiopathy after stem cell transplant. Blood Adv . Jan 12
2021;5(1):1-11. doi:10.1182/bloodadvances.2020003455
31. Laskin BL, Goebel J, Davies SM, et al. Early clinical indicators of
transplant-associated thrombotic microangiopathy in pediatric
neuroblastoma patients undergoing auto-SCT. Bone Marrow
Transplant . May 2011;46(5):682-9. doi:10.1038/bmt.2010.182