Evans syndrome after unrelated bone marrow transplantation for refractory cytopenia of childhood

Abstract

Post-transplant ES, which is often resistant to therapies, has seldom been described. This report describes a case of ES after UBMT for RCC. A five-yr-old boy developed RCC with no evidence of monosomy 7. Because no matching family donors were available for SCT and immunosuppressive therapy was ineffective, UBMT was performed when he was six yr old. The conditioning regimen included TAI (3 Gy) and administration of FLU, CY, and rabbit antithymocyte globulin. The recovery of blood cells was good. He displayed grade II acute GVHD involving only the skin. ES developed on day 66, with positive results for Epstein-Barr virus DNA and HHV 6. Cytopenia was resolved with treatment with RTX, GCV, an escalated dose of steroids, high-dose gammaglobulin, and romiplostim. No relapse has occurred since discontinuing steroids on day 177 and romiplostim on day 268. Post-SCT ES after UBMT is rare, and the risk factors and therapies are unclear. Prospective analysis and collection of cases from multiple centers are required for clarification.

Eltrombopag after allogeneic haematopoietic cell transplantation in a case of poor graft function and systematic review of the literature

“…The eight publications included observational case studies (five studies, total of seven patients) that primarily addressed idiopathic thrombocytopenia purpura (ITP) or isolated thrombocytopenia at various time points after allogeneic transplantation and prospective clinical trials (three studies, total of 177 patients with 95 patients receiving TRAs) (Tables and ). Isolated case reports described variable responses to TRA for patients with peripheral immune thrombocytopenia or amegakaryocytic thrombocytopenia following allogeneic transplant, and 6 of 7 patients had a favourable response and achieved independence from platelet transfusion within 7–23 days of treatment with romiplastim (6 patients) (DeRemer et al, ; Poon et al, ; Buchbinder et al, ; Ueki et al, ) or eltrombopag (1 patient) (Reid et al, ). Prospective studies addressed the use of TRAs to accelerate initial engraftment, either through administration starting immediately after transplant in all patients (two studies) (Liesveld et al, ; Han et al, ) or in patients with persistent thrombocytopenia at specific time points after allogeneic transplant (one study) (Nash et al, ).…”

A Review of Romiplostim Mechanism of Action and Clinical Applicability

“…There is less experience in children. [171][172][173][174][175] In particular, an Italian retrospective study recently reported on the use of eltrombopag in nine pediatric patients after HSCT; after a median treatment time of 36 days, eight of the nine patients (88%) achieved sustained platelet counts >50 × 10 9 /L. 173 In seven children with secondary failure of platelet recovery treated with romiplostim, six (86%) became transfusion-independent in the second week of treatment.…”