Since May 2007 we can treat adult primary immune thrombocytopenia (ITP) patients with the TPO receptor agonist (TPO-RA) romiplostim (clinical trials, reimbursed, medical need). From May 2007 till May 2018, 60 adult ITP patients started treatment with romiplostim at the University Hospitals of Leuven. 

The baseline characteristics of this cohort are as follows: 35 of 60 patients were male; diagnosis of primary ITP was made in 42 patients; the 17 patients with secondary ITP had a history of lymphoma (n=6), Evans syndrome (n=5), a previous liver or renal transplant (n=3) or an underlying inherited disorder (n=3); mean age  and platelet count at ITP diagnosis was 53years (y) (range 1-90y) and 16000/l; median time between ITP diagnosis and the start of romiplostim was 60months (mo) (range 0-492mo); at start of romiplostim 7 patients fulfilled the definition of acute, 7 of persistent and 45 of chronic ITP; median platelet count at start of the drug was 20000/l; 32% (19/59) were splenectomised and 39% (23/59 ) needed  3 ITP  treatments (range 3-8) before the start of the TPO-RA.

At 30 november 2018, median time on romiplostim was 21.5mo (range 2weeks -10.5y);
treatment was ongoing in 38% (n=23/60) of patients; reasons for discontinuation was non-ITP related death (n= 17), ITP related death (n=1), treatment-free remission (TFR) (n= 10), patient’s decision followed by TFR (n=2), splenectomy followed by TFR (n=2), doctor‘s decision (n=1), loss of response (n=2) and loss of follow up (n=1). Median dose of romiplostim needed for a safe platelet count was 3.75 kg. 

These data show that treatment with romiplostm is not “for all” forever. Non-ITP related death (median time on drug 20.5mo) and TFR (median time on drug 5mo) were the most common reasons of discontinuation of the drug.

Adult ITP patients treated with romiplostim in daily clinical practice: single center experience