Avatrombopag for severe refractory thrombocytopenia in a pediatric patient with ALL following allogeneic hematopoietic stem cell transplantation: A case report.

Patients who receive allogeneic hematopoietic stem cell transplantation (alloHSCT) are at risk for developing persistent thrombocytopenia. Here, we describe treatment with avatrombopag, a thrombopoietin receptor agonist, in a pediatric patient with chronic, severe, transfusion-dependent thrombocytopenia (<10 × 103/µL) post-alloHSCT that was persistent despite treatment with romiplostim, another thrombopoietin receptor agonist. Following the granting of a compassionate use investigational new drug authorization, avatrombopag treatment was initiated, and the patient's platelet count increased. To date, the patient has maintained a platelet count >100 × 103/µL. No adverse events or medication toxicities have been reported, and he has resumed his pre-alloHSCT activities.

Feasibility study of busulfan, fludarabine, and thiotepa conditioning regimen for allogeneic hematopoietic stemcell transplantationfor children and young adults with nonmalignant disorders.

BACKGROUND: Hematopoietic stem cell transplant (HSCT) is the only curative treatment for several pediatric non-malignant disorders. A widely used conditioning backbone is busulfan, fludarabine, and rabbit anti-thymocyte globulin (rATG). Thiotepa has improved engraftment when added to this regimen, however the minimum effective dose (MED) of thiotepa to achieve engraftment while minimizing toxicities has not been well established. OBJECTIVES: The primary objective of this prospective feasibility study was to determine the MED of thiotepa (5mg/kg) in combination with reduced-dose busulfan, fludarabine or cyclophosphamide, and rATG required to achieve engraftment in >90% of HSCT recipients for non-malignant disorders with acceptable toxicity. RESULTS: Six patients who received fully matched HSCT were enrolled. Patient diagnoses included Wiskott-Aldrich syndrome (n = 1), CD40L deficiency (n = 1), sickle cell disease (n = 2), autoinflammatory syndrome (n = 1), and paroxysmal nocturnal hemoglobinuria (n = 1). All six patients achieved engraftment prior to Day +42 and five patients had stable full donor engraftment. Two of the six patients (33%) developed acute GVHD and/or chronic GHVD, both of whom had sickle cell disease. At a median follow-up of 2.25 years post-transplant, all patients were alive without evidence of disease recurrence. None of the patients experienced grade 4 or 5 toxicities. Three out of six patients (50%) developed grade 3 adverse events. Neurocognitive functioning of children under 10 years of age was not adversely affected by this regimen. CONCLUSION: This approach shows acceptable toxicity and reliable engraftment in children with non-malignant disorders receiving related or unrelated HLA-matched transplants.

Radiation options for early stage breast cancer.

OBJECTIVE: To explore the current radiation therapy options for early stage breast cancer. DATA SOURCES: Research literature and review articles. CONCLUSION: With 20 years of clinical data supporting the efficacy of whole breast radiotherapy after lumpectomy for early stage breast cancer, the attention has shifted to an even more conservative approach, namely partial breast irradiation following lumpectomy. A number of large clinical trials in North America and Europe are currently enrolling patients for the evaluation of several different techniques, including interstitial brachytherapy, the Mammosite device, 3-dimensional conformal external radiation therapy, and intraoperative radiotherapy. IMPLICATIONS FOR NURSING PRACTICE: Radiation oncology nurses can significantly impact the coordination of services and the education of patients who enroll in these important clinical trials.

The role of licensed nursing personnel in radiation oncology part a: results of a descriptive study.

PURPOSE/OBJECTIVES: To identify the specific administrative, clerical, patient care, patient education, and research responsibilities that licensed nursing personnel perform in the field of radiation oncology. DESIGN: Descriptive. SAMPLE: 281 licensed nursing personnel employed in the field of radiation oncology in North America. METHODS: Subjects completed a six-page, self-administered questionnaire comprised of fixed-choice and open-ended questions. MAIN RESEARCH VARIABLES: Demographics, employment settings, and administrative, clerical, patient care, patient education, and research responsibilities. FINDINGS: Nurses in radiation oncology tend to be older (41-60 years of age) and considerably well educated, with many years of experience in this field. Nurses are responsible for a wide variety of tasks. The study found a strong demonstration of the role of nurse educator among radiation oncology nurses. IMPLICATIONS FOR NURSING: Radiation oncology nursing is a subspecialty in evolution. The data should provide support for further exploration of how patient education and support influence patient outcomes in radiation oncology.

The role of licensed nursing personnel in radiation oncology part B: integrating the ambulatory care nursing conceptual framework.

PURPOSE/OBJECTIVES: To describe the professional opportunities for licensed nursing personnel in radiation oncology within the conceptual framework developed for ambulatory care nurses by the American Academy of Ambulatory Care Nursing (AAACN). DATA SOURCES: Published articles and books. DATA SYNTHESIS: Using AAACN's framework, the researchers identified three roles that are part of every ambulatory care nurse's practice and can be readily customized to radiation oncology: an organizational/systems role, a professional role, and a clinical nursing role. Incorporating the described framework into a radiation oncology nurse's job description can lead to maximizing the use of the professional staff member within the radiation oncology department. This level of professional utilization and validation of the radiation oncology nurse will lead not only to improved patient outcomes but also to greater professional satisfaction, resulting in improved retention. CONCLUSIONS: The complexity of oncology therapies demands expert oncology nurses. This clearly is recognized in medical oncology, and patients with cancer who are being treated with radiation are no less important. To optimize patient care and therapeutic outcomes, the integration of nursing in all aspects of the radiation treatment continuum is essential. IMPLICATIONS FOR NURSING: Radiation oncology nursing is an evolving subspecialty. Nurses can use the information in this article to evaluate their current roles and individual potential for further professional growth.

Squamous cell carcinoma of the oropharynx: surgery, radiation therapy, or both.

BACKGROUND: The treatment of patients with squamous cell carcinoma (SCC) of the oropharynx remains controversial. No randomized trial has addressed adequately the question of whether surgery (S), radiation therapy (RT), or combined treatment is most effective. METHODS: Treatment results from North American academic institutions that used S with or without adjuvant RT (S +/- RT) or used RT alone or followed by neck dissection (RT +/- ND) for patients with SCC of the tonsillar region or the base of tongue were compiled through a MEDLINE search (from 1970 to August, 2000) and from the references cited in each report. Studies were eligible for inclusion if they contained direct, actuarial (life-table), or Kaplan-Meier calculations for the following end points: local control, local-regional control, 5-year absolute survival, 5-year cause specific survival, or severe or fatal treatment complications. Weighted average results, which took into account series size, were calculated for each end point for the purposes of treatment comparison. Results and conclusions were based on data from 51 reported series, representing the treatment of approximately 6400 patients from the United States and Canada. RESULTS: The results for patients with SCC of the base of tongue who underwent S +/- RT versus RT +/- ND, respectively, were as follows: local control, 79% versus 76% (P = 0.087); local-regional control, 60% versus 69% (P = 0.009); 5-year survival, 49% versus 52% (P = 0.2); 5-year cause specific survival, 62% versus 63% (P = 0.4); severe complications, 32% versus 3.8% (P < 0.001); and fatal complications, 3.5% versus 0.4% (P < 0.001). The results for patients with SCC in the tonsillar region who underwent S +/- RT versus RT +/- ND, respectively, were as follows: local control, 70% versus 68% (P = 0.2); local-regional control, 65% versus 69% (P = 0.1); 5-year survival, 47% versus 43% (P = 0.2); 5-year cause specific survival, 57% versus 59% (P = 0.3); severe complications, 23% versus 6% (P < 0.001); and fatal complications, 3.2% versus 0.8% (P < 0.001). CONCLUSIONS: The information in this article provides a useful benchmark for evidence-based counseling of patients with SCC of the oropharynx. The rates of local control, local-regional control, 5-year survival, and 5-year cause specific survival were similar for patients who underwent S +/- RT or RT +/- ND, whereas the rates of severe or fatal complications were significantly greater for the S +/- RT group. Furthermore, available data on the functional consequences of treatment suggest the superiority of RT +/- ND. The authors conclude that RT +/- ND is preferable for the majority of patients with SCC of the oropharynx.