NCCN Task Force Report: Oral chemotherapy Weingart, Saul N; Brown, Elizabeth; Bach, Peter B ...
Journal of the National Comprehensive Cancer Network,
06/2008, Letnik:
6 Suppl 3
Journal Article
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Oral chemotherapy is emerging as a new option for well-selected patients who can manage potentially complex oral regimens and self-monitor for potential complications. If a choice between oral and ...parenteral therapy is available, patients may opt for oral chemotherapy because it is more convenient to administer, allows them to avoid multiple office visits, and gives them a sense of control over their own cancer care. Whether these potential advantages are maintained in regimens that combine oral and parenteral drugs is less clear. The use of oral chemotherapeutic agents profoundly affects all aspects of oncology, including creating significant safety and adherence issues, shifting some traditional roles and responsibilities of oncologists, nurses, and pharmacists to patients and caregivers. The financing of chemotherapy is also affected. To address these issues, the NCCN convened a multidisciplinary task force consisting of oncologists, nurses, pharmacists, and payor representatives to discuss the impact of the increasing use of oral chemotherapy.
NCCN Task Force Report: Specialty Pharmacy Schwartz, Rowena N; Eng, Kirby J; Frieze, Deborah A ...
Journal of the National Comprehensive Cancer Network
8 Suppl 4
Journal Article
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The use of specialty pharmacies is expanding in oncology pharmacy practice. Specialty pharmacies provide a channel for distributing drugs that, from the payor perspective, creates economies of scale ...and streamlines the delivery of expensive drugs. Proposed goals of specialty pharmacy include optimization of pharmaceutical care outcomes through ensuring appropriate medication use and maximizing adherence, and optimization of economic outcomes through avoiding unwarranted drug expenditure. In oncology practice, specialty pharmacies have become a distribution channel for various agents. The use of a specialty pharmacy, and the addition of the pharmacist from the specialty pharmacy to the health care team, may not only provide benefits for care but also present challenges in oncology practice. The NCCN Specialty Pharmacy Task Force met to identify and examine the impact of specialty pharmacy practice on the care of people with cancer, and to provide recommendations regarding issues discussed. This report provides recommendations within the following categories: education and training of specialty pharmacy practitioners who care for individuals with cancer, coordination of care, and patient safety. Areas for further evaluation are also identified.
REMS are a particularly important issue for oncology and the National Comprehensive Cancer Network (NCCN). A disproportionate number of drugs with complex REMS are used in patients with cancer or ...hematologic disorders. REMS policies and processes within oncology may act as a model for other clinical areas. A breadth of experience and access to a wide knowledge base exists within oncology that will ensure appropriate development and consideration of the practical implications of REMS. NCCN is uniquely positioned to assume a leadership role in this process given its status as the arbiter of high-quality cancer care based on its world-leading institutions and clinicians. Notwithstanding the potential benefits, the successful design, implementation, and analysis of the FDA's recent requirement for REMS for some high-risk drugs and biologics will present significant challenges for stakeholders, including patients, providers, cancer centers, manufacturers, payors, health information technology vendors, and regulatory agencies. To provide guidance to these stakeholders regarding REMS challenges, the NCCN assembled a work group comprised of thought leaders from NCCN Member Institutions and other outside experts. The Work Group identified challenges across the REMS spectrum, including the areas of standardization, development and assessment of REMS programs, medication guides, provider knowledge and impact on prescribing, provider burden and compensation, and incorporation of REMS into clinical practice.
Specialty Pharmacy plays an integral role in the delivery and management of biologic and chemotherapeutic medications for patients with higher-risk MDS by streamlining the delivery process, educating ...patients, monitoring treatment adherence and response, managing complex storage and delivery requirements, and assisting both patients and payers with clinical, operational, and financial issues and challenges. Specialty Pharmacy will continue to develop procedures to assist clinicians and patients, maximizing access to pharmacy and clinical advisors who are able to explain medical diagnoses to patients, walk them through medication options, and describe the specific details of therapeutic regimens. Where the use of specialty pharmaceuticals poses challenges of inventory, sourcing, complex treatment regimens, patient adherence, and slow reimbursement, Specialty Pharmacy has designed standardized processes to address these needs and will continue to improve and increases its services to support vital business and clinical functions for patients, health care plans, and providers.
Whole-exome sequencing is a diagnostic approach for the identification of molecular defects in patients with suspected genetic disorders.
We developed technical, bioinformatic, interpretive, and ...validation pipelines for whole-exome sequencing in a certified clinical laboratory to identify sequence variants underlying disease phenotypes in patients.
We present data on the first 250 probands for whom referring physicians ordered whole-exome sequencing. Patients presented with a range of phenotypes suggesting potential genetic causes. Approximately 80% were children with neurologic phenotypes. Insurance coverage was similar to that for established genetic tests. We identified 86 mutated alleles that were highly likely to be causative in 62 of the 250 patients, achieving a 25% molecular diagnostic rate (95% confidence interval, 20 to 31). Among the 62 patients, 33 had autosomal dominant disease, 16 had autosomal recessive disease, and 9 had X-linked disease. A total of 4 probands received two nonoverlapping molecular diagnoses, which potentially challenged the clinical diagnosis that had been made on the basis of history and physical examination. A total of 83% of the autosomal dominant mutant alleles and 40% of the X-linked mutant alleles occurred de novo. Recurrent clinical phenotypes occurred in patients with mutations that were highly likely to be causative in the same genes and in different genes responsible for genetically heterogeneous disorders.
Whole-exome sequencing identified the underlying genetic defect in 25% of consecutive patients referred for evaluation of a possible genetic condition. (Funded by the National Human Genome Research Institute.).
To evaluate the measurement properties of the Wheelchair Use Confidence Scale for manual wheelchair users version 2.1 (WheelCon-M 2.1).
Cohort study.
Volunteer sample of 83 community-dwelling, ...experienced manual wheelchair users.
Participants completed the WheelCon-M 2.1 twice to assess retest reliability. Validity was assessed by evaluating hypothesized relationships between the WheelCon-M 2.1 and relevant variables. Responsiveness was assessed using the standard error of measurement (SEM) and smallest real difference (SRD).
The median (interquartile range) WheelCon-M 2.1 score was 84.6 (71.3-92.0) of a possible 0-100. The one-week retest intraclass correlation coefficient was 0.84 with 95% bias-corrected and accelerated confidence intervals of 0.77-0.90. Cronbach's alpha was 0.92. Correlations ranging from rs = -0.19 (p = 0.780) to rs = 0.58 (p < 0.001) were found between the WheelCon-M 2.1 and other relevant outcome measures with all correlations being statistically significant except for age (p = 0.780) and social support (p = 0.057). A statistically significant difference was not found between the sexes (p = 0.140). The SEM and SRM were 5.9 and 16.4, respectively.
WheelCon-M 2.1 has high internal consistency, strong retest reliability, and support for concurrent validity, construct validity and responsiveness. This new test holds promise as a clinical and research tool.
An extensive body of scholarship focuses on cultural diversity in health care, and this has resulted in a plethora of strategies to “manage” cultural difference. This work has often been ...patient-oriented (i.e., focused on the differences of the person being cared for), rather than relational in character. In this study, we aimed to explore how the difference was relational and coproduced in the accounts of cancer care professionals and patients with cancer who were from migrant backgrounds. Drawing on eight focus groups with 57 cancer care professionals and one-on-one interviews with 43 cancer patients from migrant backgrounds, we explore social relations, including intrusion and feelings of discomfort, moral logics of rights and obligation, and the practice of defaulting to difference. We argue, on the basis of these accounts, for the importance of approaching difference as relational and that this could lead to a more reflexive means for overcoming “differences” in therapeutic settings.
In this article, we focus on developing a critical sociology of ‘cultural and linguistic diversity’ as evident in cancer care praxis, drawing on the perspectives of cancer care health professionals. ...Set within the context of increasing efforts on the part of healthcare providers to ‘accommodate difference’ and ‘incorporate diversity’, we aimed to utilise participants’ accounts of practice to ask: how do we and how should we think about and operationalise ‘culture’ (if at all) in cancer care settings. Drawing on eight focus groups with doctors, nurses, allied health staff and multicultural community workers, here we explore their accounts of: othering and over-simplification; the role of absences in biographical reciprocity; intimacy, care and carelessness; and entanglements of culture with other aspects of the person. Based on their accounts, we argue for a broadening of the examination of the nexus of culture and care, to focus on the problematics of othering, intimacy, reciprocity and complexity.
Adaptor protein complex 1 (AP-1) is an evolutionary conserved heterotetramer that promotes vesicular trafficking between the trans-Golgi network and the endosomes. The knockout of most murine AP-1 ...complex subunits is embryonically lethal, so the identification of human disease-associated alleles has the unique potential to deliver insights into gene function. Here, we report two founder mutations (c.11T>G p.Phe4Cys and c.97C>T p.Arg33Trp) in AP1S3, the gene encoding AP-1 complex subunit σ1C, in 15 unrelated individuals with a severe autoinflammatory skin disorder known as pustular psoriasis. Because the variants are predicted to destabilize the 3D structure of the AP-1 complex, we generated AP1S3-knockdown cell lines to investigate the consequences of AP-1 deficiency in skin keratinocytes. We found that AP1S3 silencing disrupted the endosomal translocation of the innate pattern-recognition receptor TLR-3 (Toll-like receptor 3) and resulted in a marked inhibition of downstream signaling. These findings identify pustular psoriasis as an autoinflammatory phenotype caused by defects in vesicular trafficking and demonstrate a requirement of AP-1 for Toll-like receptor homeostasis.
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