Practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome in the acute phase: A review of four patients.
AIM
Recently eculizumab, a monoclonal antibody to C5, was found to improve the disease course of atypical haemolytic uraemic syndrome (aHUS) and has been recommended as the first line treatment by an international consensus guideline. However, several practical issues in the use of eculizumab for the acute phase of aHUS have yet to be resolved.


METHODS
Children who received eculizumab with diagnosis of aHUS between March 2010 and December 2015 at Tokyo Metropolitan Children's Medical Center were enrolled. aHUS was diagnosed according to the haemolytic uraemic syndrome (HUS) criteria after excluding Shiga toxin-inducing Escherichia coli (STEC) -associated HUS and thrombocytopaenic purpura. We retrieved and analyzed data from the electronic medical records at our institution.


RESULTS
We reviewed four patients with suspected aHUS. Eculizumab was discontinued in one patient in whom STEC-HUS was later diagnosed. Treatment was continued in the remaining three patients without recurrence. Practical issues included difficulty in diagnosing aHUS, particularly in the acute phase, risk of infection by encapsulated organisms, especially Neisseria meningitis, and infusion reaction. In addition to issues relating to the acute phase, discontinuing eculizumab in stable patients in the chronic phase must be considered.


CONCLUSION
Eculizumab, the first line treatment for children with aHUS, is usually effective. However, certain problems associated with its use require caution to be exercised. As clinical information on eculizumab are still very limited, and the rationale for its long-term use has yet to be established, physicians are advised to exercise care when using eculizumab to manage aHUS.
['Terano|Chikako|C|http://orcid.org/0000-0001-5033-9270', 'Ishikura|Kenji|K|http://orcid.org/0000-0003-2692-6437', 'Hamada|Riku|R|', 'Yoshida|Yasuhiro|Y|', 'Kubota|Wataru|W|', 'Okuda|Yusuke|Y|', 'Shinozuka|Shunsuke|S|', 'Harada|Ryoko|R|', 'Iyoda|Sunao|S|', 'Fujimura|Yoshihiro|Y|', 'Hamasaki|Yuko|Y|', 'Hataya|Hiroshi|H|', 'Honda|Masataka|M|']
D000367:Age Factors D061067:Antibodies, Monoclonal, Humanized D065766:Atypical Hemolytic Uremic Syndrome D002675:Child, Preschool D051056:Complement Inactivating Agents D004334:Drug Administration Schedule D003875:Drug Eruptions D057286:Electronic Health Records D005260:Female D006801:Humans D016867:Immunocompromised Host D007223:Infant D008297:Male D008589:Meningococcal Infections D009894:Opportunistic Infections D012074:Remission Induction D012189:Retrospective Studies D012307:Risk Factors D013997:Time Factors D014041:Tokyo D016896:Treatment Outcome
Eculizumab Children Atypical haemolytic uraemic syndrome Infusion reaction Meningococcal infection