Adonis Lorenzana

One year follow-up of children and adolescents with chronic immune thrombocytopenic purpura (ITP) treated with rituximab

We previously showed in a prospective study that rituximab appears to be effective in some children and adolescents with severe chronic immune thrombocytopenia. Eleven of 36 patients achieved and maintained platelet counts over 50,000/mm3 within the first 12 weeks. These patients were followed for the next year.

Physician decision making in selection of second-line treatments in immune thrombocytopenia in children

Immune thrombocytopenia (ITP) is an acquired autoimmune bleeding disorder which presents with isolated thrombocytopenia and risk of hemorrhage. While most children with ITP promptly recover with or without drug therapy, ITP is persistent or chronic in others. When needed, how to select second‐line therapies is not clear. ICON1, conducted within the Pediatric ITP Consortium of North America (ICON), is a prospective, observational, longitudinal cohort study of 120 children from 21 centers starting second‐line treatments for ITP which examined treatment decisions. Treating physicians reported reasons for selecting therapies, ranking the top three. In a propensity weighted model, the most important factors were patient/parental preference (53%) and treatment‐related factors: side effect profile (58%), long‐term toxicity (54%), ease of administration (46%), possibility of remission (45%), and perceived efficacy (30%). Physician, health system, and clinical factors rarely influenced decision‐making. Patient/parent preferences were selected as reasons more often in chronic ITP (85.7%) than in newly diagnosed (0%) or persistent ITP (14.3%, P = .003). Splenectomy and rituximab were chosen for the possibility of inducing long‐term remission (P < .001). Oral agents, such as eltrombopag and immunosuppressants, were chosen for ease of administration and expected adherence (P < .001). Physicians chose rituximab in patients with lower expected adherence (P = .017). Treatment choice showed some physician and treatment center bias. This study illustrates the complexity and many factors involved in decision‐making in selecting second‐line ITP treatments, given the absence of comparative trials. It highlights shared decision‐making and the need for well‐conducted, comparative effectiveness studies to allow for informed discussion between patients and clinicians.

Serum Levels of Soluble CD30 (sCD30) in Children 783

CD30 is a cytokine receptor that belongs to the TNF/NGF receptor superfamily. It is normally expressed in activated and virus infected T and B cells and possibly monocytes. CD30 is shed in vivo and in vitro; therefore it exists both as membrane bound and soluble forms. High serum levels of sCD30 have been found in EBV infected individuals, adult patients with Hodgkins disease and CD30 positive lymphomas (CD30+ NHL). The reported normal adult values are 5.3 ± 5.7 u/ml (mean ± SD). No information is available about normal serum levels of this receptor in children or its potential use in children with CD30+ NHL. We sought to establish the values of sCD30 in normal children using a specific ELISA method (Milenia, DPC, Germany). A total of 27 children (19 female, 8 male), age range 6 months to 16 years were studied. The sCD30 serum values ranged from 11 to 230 u/ml with a mean ± SD value of 89.5±83.25. The children were then subdivided into three groups: group 1 included 13 children who presented with normal physical exam, group 2 included 4 children who presented with URI symptoms and group 3 included 10 febrile children. The sCD30 values ranged from 11 to 44 u/ml, 51 to 230 u/ml, 30 to 230 u/ml with mean ± SD values of 28.8±9.5 u/ml, 119±83 u/ml, 156.6±82.9 u/ml for groups 1 to 3 respectively. These differences achieved statistical significance when comparing group 1 with groups 2 and 3 (p value <0.05). We studied 2 additional children with CD30 + NHL (one of them with immunodeficiency). Preliminary results indicate that sCD30 may be a marker of the disease activity. This study shows that sCD30 levels are higher in children when compared with published results in adults and that higher levels can be found in children with infections. These results may be utilized in future studies assessing the role of sCD30 levels in children with infections and CD30+ malignancies.