Paradigm shift of childhood thrombotic thrombocytopenic purpura with severe ADAMTS13 deficiency

Children and adults with thrombotic thrombocytopenic purpura associated with severe, acquired Adamts13 deficiency: Comparison of incidence, demographic and clinical features

“…In addition to the one child from the Oklahoma Registry, the systematic literature review identified 55 children <18 years old with TTP associated with severe, acquired ADAMTS13 deficiency in 24 articles (Table ) . Twenty‐three additional children <18 years old with ADAMTS13 activity <10% and a demonstrable ADAMTS13 inhibitor were identified from the records of the Central Hematology Laboratory of the Inselspital, University of Bern, Switzerland, 2002–2011.…”

“…The two children reported by Ashida and Sato were also reported by Yagi and were excluded from the Yagi data in this Table. Only one reported child was described as dying from her episode of TTP (Patient #42).…”

An international consensus approach to the management of atypical hemolytic uremic syndrome in children

“…Biological assays to confirm the clinical diagnosis of aHUS In children, clinical presentation and baseline laboratory results usually allow the diagnosis of Streptococcus pneumoniae, STEC and cobalamin C (cblC) defect-HUS or of TTP with a good degree of certainty to inform adequate treatment initiation (Table 1) [20][21][22][23][24][25][26][27][28][29][30][31][32][33][34]. Figure 2 outlines confirmatory investigations for each of the common differential diagnoses [35][36][37][38][39][40][41][42][43][44][45][46][47].…”

“…Eighty-six percent of women who have the first episode of HUS during pregnancy (mostly in the post-partum) have a complement mutation. Therefore, pregnancy-HUS is classified as alternative complement pathway dysregulation-aHUS [19] Post Congenital TTP [21][22][23] Acquired TTP [21,22,24,25] STEC-HUS [26][27][28] CblC defect-HUS [29][30][31][32][33][34] aHUS [3] Age, years Eculizumab is prescribed in some countries for patients with brain/cardiac/multivisceral involvement. Further studies are required to establish the benefit of complement blockade treatment in STEC-HUS adults [1,3].…”

Successful treatment of relapsing autoimmune thrombotic thrombocytopenic purpura with rituximab

“…in 2006 . Since then the literature on rituximab use for TTP in children has consisted mostly of individual case reports or small case series . In view of the extreme rarity of TTP in the pediatric population, the cumulative data from these publications is very important.…”