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Editorials
Figure 1. History of discovery and therapies for immune thrombocytopenia (ITP). FDA: US Food and Drug Administration.
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superior to both alternative treatments. However, given the moderate undesirable effects associated with splenec- tomy, including fatal sepsis and lifelong susceptibility to bacterial infection, TPO-RA and rituximab were pre- ferred.13
With the decline in the number of splenectomies per- formed in children with ITP, despite potential data sup- porting superior efficacy to alternative therapies and guidelines favoring newer more novel therapies, Avila et al. used the ICIS Splenectomy Registry to evaluate long- term outcomes in 239 children with ITP.1 Analysis dura- tion was a median of 25 months and included assess- ments for response and bleeding events. Interestingly, 26% of patients had splenectomies performed in the acute or persistent phase of diagnosis, which is outside the commonly recommended window per guidelines. Minimal adverse events occurred in the peri-operative window with 5% having intra-abdominal bleeding, 10% with fever, and no deaths or reports of sepsis. Of the patients followed for ≥6 months (n=168), 11% had admissions for fever and 2.7% for sepsis. Response was notable for 93% of patients achieving complete remission (CR: ≥60% of platelet counts ≥1 month post splenectomy ≥100x109/L) or remission (R: ≥60% of platelet counts ≥1 month post splenectomy ≥30x109/L). Refractoriness was seen in only 1.7% of children; however, this outcome could have been influenced by use of subsequent treat- ment. Predictors to achieve CR included older age of the
patient at the time of diagnosis, older age of the patient at the time of splenectomy, higher platelet counts in the first month following splenectomy, and a negative correlation with use of prior second-line therapy(ies).
These findings provide robust data in a large cohort of children who have undergone splenectomy for ITP, in particular with regard to the reported findings of an over 90% CR/R rate, with minimal adverse effects in the peri- operative period. Age in terms of a predictor for respon- siveness is also a novel finding for children. As suggested by the authors, this is perhaps related to the pathophysi- ology of the disease in teenagers being similar to young adults, who also have improved responsiveness to splenectomy. Although the evidence provided in this study represent novel data in support of consideration of splenectomy as opposed to other forms of second-line therapy, a number of questions remain unanswered. The reason behind the selection of splenectomy and clinical decision-making was not collected, in particular regarding the indication of treatment, e.g., treatment due to bleed- ing symptoms, disease chronicity, or other HRQoL met- rics. Splenectomy also remains the treatment of choice for emergent management of life-threatening bleeding which may be represented by a handful of cases who underwent splenectomy early in the course of their ITP in this cohort. Furthermore, the availability of alternative treatments was variable over the course of cohort enroll- ment (1997-2017), which possibly influenced selection of
haematologica | 2020; 105(11)