Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.

Background: Up-regulation of hepatic delta-aminolevulinic acid synthase 1 (ALAS1), with resultant accumulation of delta-aminolevulinic acid (ALA) and porphobilinogen, is central to the pathogenesis of acute attacks and chronic symptoms in acute hepatic porphyria. Givosiran, an RNA interference therapy, inhibits ALAS1 expression.

Methods: In this double-blind, placebo-controlled, phase 3 trial, we randomly assigned symptomatic patients with acute hepatic porphyria to receive either subcutaneous givosiran (2.

The Effect of the 2009 USPSTF breast cancer screening recommendations on breast cancer in Michigan: A longitudinal study.

In 2009, the revised United States Preventive Services Task Force (USPSTF) guidelines recommended against routine screening mammography for women age 40-49 years and against teaching self-breast examinations (SBE). The aim of this study was to analyze whether breast cancer method of presentation changed following the 2009 USPSTF screening recommendations in a large Michigan cohort. Data were collected on women with newly diagnosed stage 0-III breast cancer participating in the Michigan Breast Oncology Quality Initiative (MiBOQI) registry at 25 statewide institutions from 2006 to 2015.

Variation in the use of advanced imaging at the time of breast cancer diagnosis in a statewide registry.

Background: Although national guidelines do not recommend extent of disease imaging for patients with newly diagnosed early stage breast cancer given that the harm outweighs the benefits, high rates of testing have been documented. The 2012 Choosing Wisely guidelines specifically addressed this issue. We examined the change over time in imaging use across a statewide collaborative, as well as the reasons for performing imaging and the impact on cost of care.

Associations between use of the 21-gene recurrence score assay and chemotherapy regimen selection in a statewide registry.

Background: The 21-gene recurrence score (RS) assay predicts response to adjuvant chemotherapy in patients with early-stage, hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative invasive breast cancer, but to the authors' knowledge, the role of the assay in guiding the selection of chemotherapy regimen has not been established. The current study was conducted to examine patterns of use of the RS assay for selecting chemotherapy regimens across a statewide registry from 2006 through 2013.

Methods: Demographic, pathologic, and treatment data were abstracted from medical records for 16,666 women with breast cancer who were treated at 25 hospital systems across Michigan that were participating in the Michigan Breast Oncology Quality Initiative.

University of Michigan Comprehensive Cancer Center opportunities for improvement project.

The University of Michigan Comprehensive Cancer Center (UMCCC) Opportunities for Improvement project involved a detailed patient-level medical record review, feedback to medical providers and clinical leadership, and discussion of potential predictors of discordant or delayed care. The medical record review revealed that reasons for discordant or delayed care were well documented by clinical providers, and medical comorbidity was the most common predisposing factor. Another common theme was the difficulty in obtaining treatment records for patients who received a portion of their care outside UMCCC.

Time to adjuvant chemotherapy for breast cancer in National Comprehensive Cancer Network institutions.

Background: High-quality care must be not only appropriate but also timely. We assessed time to initiation of adjuvant chemotherapy for breast cancer as well as factors associated with delay to help identify targets for future efforts to reduce unnecessary delays.

Methods: Using data from the National Comprehensive Cancer Network (NCCN) Outcomes Database, we assessed the time from pathological diagnosis to initiation of chemotherapy (TTC) among 6622 women with stage I to stage III breast cancer diagnosed from 2003 through 2009 and treated with adjuvant chemotherapy in nine NCCN centers.

Defining Value in Cancer Care: AVBCC 2012 Steering Committee Report.

Approximately 200 oncologists, payers, employers, managed care executives, pharmacy benefit managers, and other healthcare stakeholders convened in Houston, TX, on March 28-31, 2012, for the Second Annual Conference of the Association for Value-Based Cancer Care (AVBCC). The mission of the conference was to align the various perspectives around the growing need of defining value in cancer care and developing strategies to enhance patient outcomes. The AVBCC conference presented a forum for the various viewpoints from all the stakeholders across the cancer care continuum, featuring more than 20 sessions and symposia led by nearly 30 oncology leaders.

Adoption of gene expression profile testing and association with use of chemotherapy among women with breast cancer.

Purpose: Gene expression profile (GEP) testing is a relatively new technology that offers the potential of personalized medicine to patients, yet little is known about its adoption into routine practice. One of the first commercially available GEP tests, a 21-gene profile, was developed to estimate the benefit of adjuvant chemotherapy for hormone receptor-positive breast cancer (HR-positive BC).

Patients And Methods: By using a prospective registry data set outlining the routine care provided to women diagnosed from 2006 to 2008 with HR-positive BC at 17 comprehensive and community-based cancer centers, we assessed GEP test adoption and the association between testing and chemotherapy use.

Improving breast cancer care through a regional quality collaborative.

Background: Regional collaborative organizations provide an effective structure for improving the quality of surgical care. With low complication rates and a long latency between surgical care and outcomes such as survival and local recurrence, quality measurement in breast cancer surgery is ideally suited to process measures. Diagnostic biopsy technique for breast cancer diagnosis is measurable and amenable to change at the provider level.

Reassessments of ESAs for cancer treatment in the US and Europe.

Anemia is a widely prevalent complication among cancer patients. At the time of diagnosis, 30% to 40% of patients with non-Hodgkin lymphoma or Hodgkin lymphoma and up to 70% of patients with multiple myeloma are anemic; rates are higher among persons with myelodysplastic syndromes. Among patients with solid cancers or lymphomas, up to half develop anemia following chemotherapy.

Impending challenges in the hematopoietic stem cell transplantation physician workforce.

With increasing use of high dose chemotherapy with autologous and allogeneic transplants the need for the transplant physician workforce requires reassessment. The types of transplants and patients are also shifting toward transplants being done in patients with more comorbidities and more commonly these types of patients require more work effort per patient from the transplant physician. Additionally, HSCT survivors often require ongoing care at the transplant center due to the inability of the primary care workforce or the hematology/oncology workforce to absorb caring for post complex post transplant patients.

Erythropoietin-stimulating agents in oncology.

Erythropoiesis stimulating agents (ESAs) are some of the most widely used agents in oncology. Yet, the use of ESAs to treat chemotherapy induced anemia in cancer patients has raised issues of concern since 2003, when the two initial studies that were powered to detect meaningful differences in survival with ESA therapy were either halted early, or concluded with deleterious effects on survival. Several trials since then have caused both guideline writers and the FDA to recommend that ESAs should not employed to drive the hemoglobin to greater than 12 g/dL.

Venous thromboembolism and mortality associated with recombinant erythropoietin and darbepoetin administration for the treatment of cancer-associated anemia.

Context: The erythropoiesis-stimulating agents (ESAs) erythropoietin and darbepoetin are licensed to treat chemotherapy-associated anemia in patients with nonmyeloid malignancies. Although systematic overviews of trials have identified venous thromboembolism (VTE) risks, none have identified mortality risks with ESAs.

Objective: To evaluate VTE and mortality rates associated with ESA administration for the treatment of anemia among patients with cancer.

Unrelated donor bone marrow transplantation using a chemotherapy-only preparative regimen for adults with high-risk acute myelogenous leukemia.

Limited data are available for adults undergoing unrelated donor (URD) BMT for AML using chemotherapy-only preparative regimens. Previous studies incorporated irradiation, included adults and children, and excluded secondary leukemia. Herein we report long-term outcomes for adults with poor-prognostic AML receiving a novel regimen of busulfan (16 mg/kg), cytarabine (8,000 mg/m(2)), and cyclophosphamide (120 mg/kg) (BAC), followed by URD BMT.

Phase I study of involved-field radiotherapy preceding autologous stem cell transplantation for patients with high-risk lymphoma or Hodgkin's disease.

Purpose: This Phase I study was designed to evaluate the tolerability of involved-field radiotherapy (IFRT) to areas of persistent disease in patients with high-risk Hodgkin's disease and non-Hodgkin's lymphomas before autologous stem cell transplantation (ASCT).

Methods And Materials: Thirty-one patients with primary refractory or relapsed Hodgkin's disease (n = 13) and non-Hodgkin's lymphoma (n = 18) were treated with IFRT followed by high-dose chemotherapy and ASCT. All patients had bulky disease (> or =5 cm) and/or an inadequate response to salvage chemotherapy.