Hematopoietic stem cell transplantation-associated thrombotic microangiopathy accompanied by renal arteriolar C4d deposition.
HSCT-associated thrombotic microangiopathy (TA-TMA) is a severe complication with a poor prognosis. Recently, it has been reported that complement system dysregulation, such as CFH autoantibodies and deletions CFH-related genes 3 and 1, induced TA-TMA. In addition, C4d-positive renal arterioles are both a good marker of complement system activation and a useful diagnostic tool for TA-TMA. Because dysregulation of the complement system is associated with TA-TMA, the complement system might be a therapeutic target, such as eculizumab, a terminal complement inhibitor. Herein, we describe an eight-yr-old boy who developed TA-TMA accompanied by severe renal dysfunction. His renal specimen showed diffuse C4d deposition in the renal arterioles, which is consistent with TA-TMA. Although the patient gradually improved without eculizumab, renal arteriolar C4d staining in sample with TA-TMA shows the complement system activation and may guide the target therapy using the eculizumab.
Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kyoto, Japan.
Kenichi
K
Sakamoto
Toshihiko
T
Imamura
Shinya
S
Osone
Ayako
A
Nishimura
Masashi
M
Nishida
Hiroyuki
H
Ishida
Hajime
H
Hosoi
D061067:Antibodies, Monoclonal, Humanized / Q000627:therapeutic use; D015415:Biomarkers; D001706:Biopsy; D002648:Child; D015935:Complement C4b / Q000737:chemistry*; D051056:Complement Inactivating Agents / Q000737:chemistry; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007668:Kidney / Q000276:immunology; D015470:Leukemia, Myeloid, Acute / Q000209:etiology; D008297:Male; D010446:Peptide Fragments / Q000737:chemistry*; D015452:Precursor B-Cell Lymphoblastic Leukemia-Lymphoma / Q000628:therapy; D011379:Prognosis; D012074:Remission Induction; D012077:Renal Artery / Q000378:metabolism*; D057049:Thrombotic Microangiopathies / Q000209:etiology* / Q000276:immunology; D016896:Treatment Outcome
D061067:Antibodies, Monoclonal, Humanized; D015415:Biomarkers; D051056:Complement Inactivating Agents; D010446:Peptide Fragments; D015935:Complement C4b; C032261:complement C4d; C481642:eculizumab
C4d deposition; complements system; hematopoietic stem cell transplantation; thrombotic microangiopathy