Need for aligning the definition and reporting of cytokine release syndrome (CRS) in immuno-oncology clinical trials.

As cancer immunotherapies continue to expand across all areas of oncology, it is imperative to establish a standardized approach for defining and capturing clinically important toxicities, such as cytokine release syndrome (CRS). In this paper, we provide considerations for categorizing the variety of adverse events that may accompany CRS and for recognizing that presentations of CRS may differ among various immunotherapies (e.g.

Paediatric Strategy Forum for medicinal product development of chimeric antigen receptor T-cells in children and adolescents with cancer: ACCELERATE in collaboration with the European Medicines Agency with participation of the Food and Drug Administration.

The seventh multi-stakeholder Paediatric Strategy Forum focused on chimeric antigen receptor (CAR) T-cells for children and adolescents with cancer. The development of CAR T-cells for patients with haematological malignancies, especially B-cell precursor acute lymphoblastic leukaemia (BCP-ALL), has been spectacular. However, currently, there are scientific, clinical and logistical challenges for use of CAR T-cells in BCP-ALL and other paediatric malignancies, particularly in acute myeloid leukaemia (AML), lymphomas and solid tumours.

ACCELERATE and European Medicine Agency Paediatric Strategy Forum for medicinal product development for mature B-cell malignancies in children.

Paediatric Strategy Forums have been created by the multistakeholder organisation, ACCELERATE, and the European Medicines Agency to facilitate dialogue between all relevant stakeholders and suggest strategies in critical areas of paediatric oncology drug development. As there are many medicines being developed for B-cell malignancies in adults but comparatively few in children with these malignancies, a Paediatric Strategy Forum was held to discuss the best approach to develop these products for children. It was concluded that as current frontline therapy is highly successful, despite associated acute toxicity, de-escalation of this or substitution of presently used drugs with new medicines can only be undertaken when there is an effective salvage regimen, which is currently not available.

Is dedicated chest CT needed in addition to PET/CT for evaluation of pediatric oncology patients?

Purpose: To assess the computed tomography (CT) portion of a positron emission tomography (PET)/CT, at lower dose without breath holding, as compared to diagnostic chest CT (dCTC), performed at regular dose with breath holding, and question the necessity of both for patient care in pediatric oncology.

Materials And Methods: This retrospective study included 46 pediatric patients with histologically proven malignant tumors that had a total of 119 scans.

Results: A total of 29 discrepancies were found between dCTC and PET/CT reports.

Connect MM Registry: The Importance of Establishing Baseline Disease Characteristics.

Background: Connect MM is the first and largest observational, noninterventional, prospective registry of patients newly diagnosed with multiple myeloma (NDMM) in the United States. It collects longitudinal data on patients within clinical practice including patients in clinical trials.

Patients And Methods: Of the 1513 patients enrolled, 1493 were protocol-eligible.

Ten-year follow-up of pediatric patients with non-Hodgkin lymphoma treated with allogeneic or autologous stem cell transplantation.

Background: Autologous or allogeneic hematopoietic stem cell transplant (SCT) is often considered in patients with relapsed or refractory non-Hodgkin lymphoma (NHL) but there are limited data on the use of SCT for the treatment of NHL in the pediatric setting.

Procedure: To evaluate the role of SCT for children with NHL, we reviewed 36 consecutive pediatric patients with NHL who underwent an allogeneic (n = 21) or autologous (n = 15) SCT at our institution between 1982 and 2004. Pathologic classification included: lymphoblastic lymphoma (n = 12), Burkitt lymphoma (BL) (n = 5), diffuse large B-cell lymphoma (n = 4), anaplastic large cell lymphoma (ALCL) (n = 13), peripheral T cell lymphoma (n = 1), and undifferentiated NHL (n = 1).

Imaging findings of ecthyma gangrenosum, an unusual complication of pseudomonas sepsis.

A child with acute lymphoblastic leukemia and pseudomonas sepsis rapidly developed left foot pain and swelling. A diagnosis of ecthyma gangrenosum was made. The clinical and imaging features of this unusual entity are discussed.

Stem cell transplant for children with sickle cell anemia: parent and patient interest.

We describe adolescents' and parents' interest in hematopoietic stem cell transplant (HSCT) as a cure for sickle cell disease (SCD) and factors associated with increased interest. We administered a 40 question survey to assess the interest in HSCT in parents and adolescents with HBSS or HBSβ(0) thalassemia. The survey tool assessed factors that may influence interest in HSCT including demographic data, disease severity, views on prognosis, and health-related quality of life (HRQOL).

Hepatoblastoma in a mosaic trisomy 18 patient.

We report a case of hepatoblastoma in a 10-year-old girl with mosaic-type trisomy 18. A comprehensive literature review reveals only 2 cases involving mosaic trisomy 18 patients. Our patient underwent an abbreviated chemotherapy course before complete surgical resection.

Dynamics of cell-mediated immune responses to cytomegalovirus in pediatric transplantation recipients.

CMI responses, combined with quantification of CMV DNA (DNAemia), may identify transplantation recipients at risk for invasive disease. PBMC were collected in pediatric transplantation candidates at one, three, and six months post-transplant in 10 subjects (six renal, three cardiac, one stem cell) and at single time points in eight HC and 14 children greater than one yr post-transplant (LTTx). Cells were stimulated with anti-CD3mAb or CMV pp65 peptide pools and responses assessed by IFNG enzyme-linked immunosorbent spot assay and cytokine secretion.