Atezolizumab for Advanced Alveolar Soft Part Sarcoma.

Background: Alveolar soft part sarcoma (ASPS) is a rare soft-tissue sarcoma with a poor prognosis and no established therapy. Recently, encouraging responses to immune checkpoint inhibitors have been reported.

Methods: We conducted an investigator-initiated, multicenter, single-group, phase 2 study of the anti-programmed death ligand 1 (PD-L1) agent atezolizumab in adult and pediatric patients with advanced ASPS.

Pediatric Chronic Myelogenous Leukemia in T-lineage Blast Crisis: A Reminder in Relevance.

Pediatric Philadelphia chromosome positive (Ph+) acute T-cell lymphoblastic leukemia can mimic chronic myelogenous leukemia (CML) in T-lineage blast crisis (BC). Differentiating the 2 is critical in guiding therapy as most children with de novo Ph+ acute T-cell lymphoblastic leukemia are treated with chemotherapy and tyrosine kinase inhibitors, whereas T-lineage BC of CML can include hematopoietic stem cell transplantation. We present a unique case of CML in T-lineage BC.

Physician approaches to drug shortages: Results of a national survey of pediatric hematologist/oncologists.

Aim: To evaluate personnel involved in scarce drug prioritization and distribution and the criteria used to inform drug distribution during times of shortage among pediatric hematologists/oncologists.

Methods: Using the American Society of Pediatric Hematology/Oncology (ASPHO) membership list, a 20 question survey of pediatric hematologists/oncologists was conducted email to evaluate personnel involved in scarce drug prioritization and distribution and criteria used to inform scarce drug distribution.

Results: Nearly 65% of the 191 study respondents had patients directly affected by drug shortages.

An ethical framework for responding to drug shortages in pediatric oncology.

The frequency of drug shortages has increased considerably over the last decade. Important ethical issues arise whenever the supply of an effective drug is insufficient to meet demand. Using the ethical principles of beneficence, non-maleficence, and justice, institutions can guide prioritization of drug distribution before a shortage occurs to avoid unfair and unethical distribution of resources.

Liver transplant for congenital factor VII deficiency.

Congenital factor VII (FVII) deficiency is a rare, autosomal recessive bleeding disorder with a spectrum of phenotypes ranging from asymptomatic to life-threatening intra-cranial hemorrhage (ICH). Orthotopic liver transplantation has been described for definitive treatment in a few patients with severe manifestations. We report a patient with congenital FVII deficiency and recurrent ICH, despite twice-weekly prophylaxis with recombinant activated FVII.

Improving pediatric hematology/oncology care in the emergency department.

Purpose: Pediatric hematology/oncology patients frequently use the emergency department (ED) for prompt care during potentially life-threatening events, such as sepsis and bleeding. One challenge of these visits is the unavailability of appropriate patient-specific medical information. Lack of information may result in ineffective ED visits for these patients with complex conditions.

Impact of cytomegalovirus (CMV) reactivation after umbilical cord blood transplantation.

This study investigated the impact of pretransplant cytomegalovirus (CMV) serostatus and posttransplant CMV reactivation and disease on umbilical cord blood transplant (UCBT) outcomes. Between 1994 and 2007, 332 patients with hematologic malignancies underwent UCBT and 54% were CMV seropositive. Pretransplant recipient CMV serostatus had no impact on acute or chronic graft-versus-host disease (aGVHD, cGVHD), relapse, disease-free survival (DFS), or overall survival (OS).

Cutaneous leishmaniasis with boggy induration and simultaneous mucosal disease.

A woman had cutaneous, mucosal, and possible visceral leishmaniasis simultaneously. Many of her cutaneous lesions consisted of boggy indurations rather than customary papules, nodules, or ulcers. This unusual case was finally cured after four courses of miltefosine, one course of antimony, and two courses of Ambisome.