@entity618 and @entity3446 as second or further line treatment in @entity1 with chronic refractory Primitive or Secondary XXXX : a single centre experience.

@entity1 @entity8782 @entity1503 @entity3446 @entity2098 @entity618 @entity230 @entity792
UNASSIGNED: The management of refractory @entity792 in childhood is challenging due to the lack of established evidence on escalating treatments. The long-term efficacy of immunosuppressive drugs was evaluated in @entity1 with refractory @entity792 referred to the Haematology Unit of the Gaslini @entity1 's Hospital between 2001 and 2014. @entity1 were grouped into three categories: @entity8782 ( @entity8782 ), @entity8782 (at least one absolute/primary additional criterion for @entity8782 ) and primary @entity792 (PAC, @entity1503 with no other immunological symptoms/signs). Fifty-eight @entity1 (aged 1-16  years) entered the study: 12 were categorized with @entity8782 , 24 were @entity8782 -related and 22 had PAC. Five didn't receive treatment. Fifty-three were initially treated with @entity230 /intravenous immunoglobulin. Fourteen responded, whereas 39 did not. Of these 39 @entity1 , 34 (87%) received @entity618 ( @entity2098 ) as second/further-line treatment and 22 (65%) responded. Within these 34 subjects, @entity8782 @entity1 responded better (11/11, 100%) than the two other groups pooled together (11/23, 48%; P  =  0  002). @entity3446 was given as second/further-line treatment to 16 @entity1 , and 12 (75%) responded, including 8 who previously failed @entity2098 therapy. Median follow-up was 3  46  years. @entity2098 and @entity3446 were well-tolerated and enabled partial/complete and sustained remission in most @entity1 . These drugs may be successfully and safely used in @entity1 with refractory @entity792 with or without @entity8782 / @entity8782 and may represent a valid second/further line option.
