Pharmacokinetics, pharmacodynamics, safety, and efficacy of crizanlizumab in patients with sickle cell disease: final results from the phase II SOLACE-adults study.

Background: Crizanlizumab is a novel inhibitor of P-selectin, a key player in multicellular adhesion and inflammatory signaling, that leads to vaso-occlusion in sickle cell disease (SCD).

Objectives: The SOLACE-adults study evaluated the pharmacokinetics, pharmacodynamics (P-selectin inhibition), safety, and efficacy of crizanlizumab, with or without hydroxyurea/hydroxycarbamide, in patients with SCD.

Design: Phase II, single-arm, multicenter study.

Efficacy of post-induction therapy for high-risk neuroblastoma patients with end-induction residual disease.

Background: High-risk neuroblastoma patients with end-induction residual disease commonly receive post-induction therapy in an effort to increase survival by improving the response before autologous stem cell transplantation (ASCT). The authors conducted a multicenter, retrospective study to investigate the efficacy of this approach.

Methods: Patients diagnosed between 2008 and 2018 without progressive disease with a partial response or worse at end-induction were stratified according to the post-induction treatment: 1) no additional therapy before ASCT (cohort 1), 2) post-induction "bridge" therapy before ASCT (cohort 2), and 3) post-induction therapy without ASCT (cohort 3).

The addition of autologous stem cell transplantation to neoadjuvant chemotherapy, radiation, and HIPEC for patients with unresectable desmoplastic small round cell tumor: a single center case series.

Desmoplastic small round cell tumor (DSRCT) is a rare, highly aggressive malignancy primarily affecting children and young adults. Although modest improvements have been gained by intensification of chemotherapy and radiation, survival of patients with DSRCT remains poor, particularly in those with unresectable or disseminated disease. We report 3 pediatric patients who were treated with a combination of therapy including chemotherapy, surgical debulking, hyperthermic intraperitoneal chemotherapy, whole abdominal irradiation, and autologous hematopoietic stem cell transplantation following busulfan and melphalan conditioning.

Immune Reconstitution Following Autologous Stem Cell Transplantation in Patients with High-Risk Neuroblastoma at the Time of Immunotherapy.

Outcomes for patients with high-risk neuroblastoma (HR-NBL) are significantly improved with the addition of immunotherapy (dinutuximab + cytokines) following autologous hematopoietic stem cell transplantation (auto-HSCT). We hypothesized that the immune system is not fully reconstituted at the initiation of immunotherapy. To test this hypothesis, we evaluated hematologic and immune subsets in 34 patients with HR-NBL before and after auto-HSCT.

Killing Two Cells with One Stone: Pharmacologic BCL-2 Family Targeting for Cancer Cell Death and Immune Modulation.

A crucial component of regulating organismal homeostasis is maintaining proper cell number and eliminating damaged or potentially malignant cells. Apoptosis, or programed cell death, is the mechanism responsible for this equilibrium. The intrinsic apoptotic pathway is also especially important in the development and maintenance of the immune system.

Irreversible electroporation in the curative treatment of Ewing's sarcoma.

Ewing's sarcoma (ES) is the second most common paediatric cancer of the bone. Standard therapy includes surgery or radiation for local control of primary and relapsed lesions and chemotherapy for systemic control. Irreversible electroporation (IRE), which uses short electrical pulses to induce pores and ablate neoplastic cells, has emerged as an alternative method of local control for inoperable metastatic liver and lung lesions.

Anemia in the Neonate: The Differential Diagnosis and Treatment.

Anemia is a common problem in the neonatal period. Presenting symptoms may suggest numerous possible diagnoses ranging from anemia seen as a normal part of development to anemia due to critical pathology. An illustrative case is presented to highlight the appropriate evaluation of the neonate with significant anemia.

Family Refusal of Chemotherapy for Pediatric Cancer Patients: A National Survey of Oncologists.

Background: Refusal of therapy is ethically acceptable for competent adults. Practitioner opinions regarding refusal of therapy in pediatric cancer patients has not been widely studied. This is the largest survey of oncology practitioners assessing support for refusal of chemotherapy.

Cytogenetic and molecular study of the PRDX4 gene in a t(X;18)(p22;q23): a cautionary tale.

The PRDX4 gene located at Xp22 encodes for a member of the peroxiredoxin gene family. Genes within this family exhibit thioredoxin-dependent peroxidase activity and have been implicated in cellular functioning, including proliferation and differentiation. Recently, PRDX4 has been identified as a partner gene in a t(X;21) translocation in a patient with acute myeloid leukemia.