Why do we overtreat children with immune thrombocytopenia despite existing evidence-based guidelines?

This issue of the Journal features an article that presents the results of a prospective study on the adherence of specialised Italian paediatric haematology centres to international and national guidelines for the treatment of children with newly diagnosed immune thrombocytopenia (ITP)¹. A clear tendency towards overtreatment is shown, which contradicts the guidelines’ recommendations. It also becomes clear that, in deviation from the guidelines, invasive diagnostics (bone marrow biopsy) is sometimes carried out. A number of non-evident influencing factors for the therapeutic decision are mentioned. This phenomenon (the deviation from evidence-based recommendations) has been the subject of much discussion^2,3, with paediatricians consistently making an important contribution, in particular with respect to vaccination issues. There are also complaints about deviations from the recommendations regarding the treatment of newly diagnosed ITP in adults⁴. In the past, guidelines often used to be expert opinions with a low level of evidence, given in the style of “old men sitting around a table, giving recommendations”. Today, however, they are often prepared with the help of methodologists and after extensive research, and therefore exhibit a high degree of evidence. The often-used argument of legal reasons for a divergent therapy decision is therefore not valid, since reviewers are expected to take the available evidence into account before giving judgement. More than 20 years ago, Pathman developed a model that allows for a theoretical analysis of the reasons for non-adherence⁵. This model describes the implementation of guidelines in four steps: awareness, agreement, adoption, and adherence. In a retrospective study in Germany (Wiegering V and Eberl W, 2020, unpublished data), we have recently found results similar to those of the authors. We interviewed 117 paediatric haematologists with a case vignette. Eight-five percent of the completed questionnaires came from 46 level 3 university or community hospitals. In deviation from the actual German guideline⁶, bone marrow biopsy is carried out in 30% of children with a bleeding score of 2, and therapy with steroids or high dose of immunoglobulin is done in 43% of the cases. Platelet transfusions are proposed in 6% of the institutions. So far, however, we have not analysed the reasons for the deviation. However, in the course of updating the guidelines^6,7, we have discussed whether the discrepancy should make us weaken our recommendations with respect to a restrictive therapy indication. But it is our view that this would have undermined the character of an evidence-based guideline. The alternative would be to analyse the reasons for the non-adherence and to try to reduce the obstacles. A thorough analysis of this issue has already been carried out⁸. The following aspects are, in our opinion, relevant to improve acceptance of our guidelines.

• The recommendations must be clearly formulated and easy to understand.

• The information on disease, symptoms, risks, course of the disease, therapy effects and adverse effects must be made accessible to the patients and their families in various forms and media.

• The guideline must be published and repeatedly made available at conferences, in specialist journals, and also within the general paediatric community.

• Adherence should be monitored at regular intervals, and the results should be communicated in an appropriate form.

In the end, it is we the doctors who talk to the patients and their parents. The quality of our consultations and our own convictions will have a crucial influence on the result; in this case, the decision for or against a particular therapy.