Practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome in the acute phase: A review of four patients

被引:4
作者
Terano, Chikako [1 ]
Ishikura, Kenji [1 ,2 ]
Hamada, Riku [1 ]
Yoshida, Yasuhiro [3 ]
Kubota, Wataru [1 ]
Okuda, Yusuke [4 ]
Shinozuka, Shunsuke [5 ]
Harada, Ryoko [1 ]
Iyoda, Sunao [6 ]
Fujimura, Yoshihiro [7 ]
Hamasaki, Yuko [1 ,8 ]
Hataya, Hiroshi [1 ]
Honda, Masataka [1 ]
机构
[1] Tokyo Metropolitan Childrens Med Ctr, Dept Nephrol, Fuchu, Tokyo, Japan
[2] Natl Ctr Child Hlth & Dev, Div Nephrol & Rheumatol, Tokyo, Japan
[3] Univ Tokyo, Grad Sch Med, Dept Pediat, Tokyo, Japan
[4] Shiga Univ Med Sci, Dept Pediat, Otsu, Shiga, Japan
[5] Natl Hosp Org Saitama Natl Hosp, Dept Pediat, Saitama, Japan
[6] Natl Inst Infect Dis, Dept Bacteriol 1, Tokyo, Japan
[7] Nara Med Univ, Dept Blood Transfus Med, Kashihara, Nara, Japan
[8] Toho Univ, Dept Pediat Nephrol, Fac Med, Tokyo, Japan
关键词
Atypical haemolytic uraemic syndrome; Children; Eculizumab; Infusion reaction; Meningococcal infection;
D O I
10.1111/nep.13054
中图分类号
R5 [内科学]; R69 [泌尿科学(泌尿生殖系疾病)];
学科分类号
1002 ; 100201 ;
摘要
AimRecently 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. MethodsChildren 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. ResultsWe 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. ConclusionEculizumab, 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. Summary at a Glance This manuscript describes practical issues in using eculizumab for children with atypical haemolytic uraemic syndrome (aHUS). Since clinical information on the use of eculizumab for children with aHUS are limited, and the rationale for its long-term use has yet to be established, this study would provide helpful information for clinicians.
引用
收藏
页码:539 / 545
页数:7
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