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Neuroblastoma (NB) is the most frequent pediatric extracranial solid tumor characterized by extreme biological heterogeneity with variable clinical course. Older age is an important risk factor. These patients may lack other common risk features but still have a chemoresistant disease with dismal prognosis. As there is currently no consensus on optimal treatment for patients with primary resistant NB, a number of clinical options is being explored including immunotherapy-based approaches. Immunotherapy with dinutuximab beta (DB) have proven its effectiveness as maintenance therapy. Allogeneic stem cell transplantation from haploidentical donor (haplo-HSCT) may be an effective consolidation in some cases. However, all forms of immunotherapy are much less effective in patients with large residual tumor. While there is no data on immune checkpoints inhibitors effectiveness in NB, some patients may benefit from this option as a part of complex immunotherapy strategy. Case presentation A 12-year old girl with gross paravertebral thoracic and abdominal tumor was diagnosed with undifferentiated neuroblastoma and bone metastases. While there was no response to several lines of chemotherapy, and only partial tumor resection was possible, the hematopoietic stem cell transplantation from haploidentical donor (haplo-HSCT) was performed as salvage therapy. Since there was only minor decrease in tumor volume with good dynamics by MIBG scan, additional post-transplant therapy was initiated. External beam radiotherapy was given for local control. The patient also received combined immunotherapy with DB and nivolumab. Currently, 3.5 years post haplo-HSCT, despite still gross residual tumor mass, it is MIBG-negative and shows signs of differentiation. Conclusion The combination of haplo-HSCT with post-transplant anti-GD2 and nivolumab may lead to a long-term response in an adolescent with primary resistant NB in spite of a large residual tumor mass.

Immune checkpoint inhibitors (ICIs) are rather efficient in classical Hodgkin's lymphoma (cHL). Pembrolizumab (pembro) is approved in children and demonstrates high response rates with acceptable toxicity. The role of nivolumab (nivo) in pediatric cHL is only to be elucidated. The aim of the presented study was to assess safety and efficiency of nivo in this age group with relapsed or refractory (R-R) cHL. Twenty-one pediatric heavily pre-treated patients 9-18 years old received nivo-based therapy. Overall response was registered in 86% (complete response-57% and partial response-29%). Three-year overall survival (OS) and progression free survival (PFS) were 95% and 29%, respectively. Only 1 clinically significant adverse effect (AE) of nivo was registered in the study (autoimmune thyroiditis). We did not observe any unacceptable toxicity of nivo.

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Please indicate where the abstract has been published before: The Lancet Infectious Diseases Background: Mucormycosis is a rare disease that may progress rapidly. Although urgent surgical and medical intervention is lifesaving, diagnosis is often delayed and mortality rates are high. Guidance on the complex multidisciplinary management has the potential to improve prognosis. Management pathways depend on recognising disease patterns and availability of diagnostic and therapeutic options. Aims: To address differences between the regions of the world, authors from 33 countries analysed published evidence on mucormycosis management and provide recommendations as part of the "One World One Guideline'' initiative. Methods: Consensus process involving experts from all UN regions, comprising physicians and scientists involved in various aspects of mucormycosis management, representing the fields of microbiology, pathology, radiology, infectious diseases, surgery, paediatrics, haematology, intensive care, dermatology, and pharmacology. Results: This guidance document provides practical help in clinical decision making and identifies areas of uncertainty and future research direction. Summary/Conclusion: Mucormycosis requires different tailored approaches in different regions of the world-This guidance document is the first in a series from One World One Guideline initiative.