The burden of managing pleural effusions in patients with chronic myelogenous leukemia post-imatinib failure: A literature-based economic analysis

Jennifer Stephens, Kimbach Tran Carpiuc, Marc BottemanPharmerit North America LLC, Bethesda, MD, USAObjectives: To develop an economic analysis of the management of pleural effusions in patients with imatinib-resistant/intolerant chronic myelogenous leukemia (CML).Methods: A cost of treatment analysis was conducted from the US payer perspective, based on resource utilization data for 48 patients with dasatinib-related pleural effusions at a large US cancer center. Probabilities of various procedures and treatment events were derived from published resource use data, supplemented with expert opinion. Cost data was derived from median reimbursements for relevant CPT codes for outpatient services and medical literature for inpatient services. Sensitivity analyses were conducted for types of procedures used. All costs were adjusted to US dollars (2007 rates).Results: Sixty percent of pleural effusions were managed medically costing 750 per episode. Forty percent of pleural effusions were more significant (>25% of one lung volume), with half of those requiring invasive procedures. Cost of inpatient procedures was 10,616 for chest tube and $15,170 with pleural catheter. Cost of outpatient procedures was$713 for ultrasound thoracentesis and $4,598 for pleural catheter. The average cost of treating a pleural effusion was$2,062 to >$2,700 for all severity levels and ~$6,400 to >$9,000 for invasive procedures. Key cost drivers were invasive procedures and recurrence.Conclusion: This economic analysis using actually observed treatment patterns suggests that the management of pleural effusion adverse events in CML patients is costly, requires intensive resource utilization, and may be an important factor in treatment selection.Keywords: dasatinib, nilotinib, imatinib, adverse events, safety, cost, and cost analysi

Indirect comparisons of second-generation tyrosine kinase inhibitors in CML: case study using baseline population characteristics

The use of indirect comparisons to evaluate the relative effectiveness between two or more treatments is widespread in the literature and continues to grow each year. Appropriate methodologies will be essential for integrating data from various published clinical trials into a systematic framework as part of the increasing emphasis on comparative effectiveness research. This article provides a case study example for clinicians using the baseline study population characteristics and response rates of the tyrosine kinase inhibitors in imatinibresistant or imatinib-intolerant chronic myelogenous leukemia followed by a discussion of indirect comparison methods that are being increasingly implemented to address challenges with these types of comparisons

An appraisal of childhood vision problems : : evaluating risk factors and the psychosocial impact of strabismus in children

Strabismus is an anomaly of ocular alignment, resulting in a departure from the parallel nature of a normal gaze, and affects approximately 1-5% of the population. Left uncorrected, strabismus can result in amblyopia, a permanent vision loss in an otherwise normal eye. Although strabismus can happen at any age, the peak age of onset is around 3 years. Despite being a relatively common eye problem in young children, little attention has been given to strabismus in the published literature, with limited research exploring the etiology of strabismus. This dissertation was undertaken with the goal of better understanding factors associated with the development of strabismus, and to review the psychosocial and health- related quality of life (HRQoL) implications of having strabismus in young children. Using data from the Child Health and Development Studies (CHDS), in-utero exposure to maternal smoking, alcohol, and coffee during pregnancy, as well as multiple perinatal factors, were explored as possible risk factors with the association of esotropia and exotropia, the most common forms of strabismus in children. Overall, the risk of developing esotropia or exotropia were increased in children whose mothers smoked heavily during pregnancy compared to those who did not, after controlling for covariates. No association was found in children whose mothers consumed alcohol or coffee during pregnancy compared to those who did not drink; perinatal factors, including maternal hemorrhage before delivery, delivery position, type of delivery, fetal stress, head circumference, and baby cry time, were also not found to be significantly associated with an increased risk of strabismus in this study population. The literature review highlights concerns related to self- image, social and school phobias, mental-health problems, and interpersonal relationships in children with strabismus, as well as strabismus-related distress in caregivers. Studies involving children have only recently found their place in quality of life research, with few studies examining the impact of HRQoL from strabismus or its treatments. The work of this dissertation adds to the small yet growing field of pediatric ophthalmology and strabismus. Additional research is needed to better understand the causes of strabismus, as well as the overall quality of life burden of strabismus in childre

An appraisal of childhood vision problems : : evaluating risk factors and the psychosocial impact of strabismus in children

Strabismus is an anomaly of ocular alignment, resulting in a departure from the parallel nature of a normal gaze, and affects approximately 1-5% of the population. Left uncorrected, strabismus can result in amblyopia, a permanent vision loss in an otherwise normal eye. Although strabismus can happen at any age, the peak age of onset is around 3 years. Despite being a relatively common eye problem in young children, little attention has been given to strabismus in the published literature, with limited research exploring the etiology of strabismus. This dissertation was undertaken with the goal of better understanding factors associated with the development of strabismus, and to review the psychosocial and health- related quality of life (HRQoL) implications of having strabismus in young children. Using data from the Child Health and Development Studies (CHDS), in-utero exposure to maternal smoking, alcohol, and coffee during pregnancy, as well as multiple perinatal factors, were explored as possible risk factors with the association of esotropia and exotropia, the most common forms of strabismus in children. Overall, the risk of developing esotropia or exotropia were increased in children whose mothers smoked heavily during pregnancy compared to those who did not, after controlling for covariates. No association was found in children whose mothers consumed alcohol or coffee during pregnancy compared to those who did not drink; perinatal factors, including maternal hemorrhage before delivery, delivery position, type of delivery, fetal stress, head circumference, and baby cry time, were also not found to be significantly associated with an increased risk of strabismus in this study population. The literature review highlights concerns related to self- image, social and school phobias, mental-health problems, and interpersonal relationships in children with strabismus, as well as strabismus-related distress in caregivers. Studies involving children have only recently found their place in quality of life research, with few studies examining the impact of HRQoL from strabismus or its treatments. The work of this dissertation adds to the small yet growing field of pediatric ophthalmology and strabismus. Additional research is needed to better understand the causes of strabismus, as well as the overall quality of life burden of strabismus in childre

Indirect comparisons of second-generation tyrosine kinase inhibitors in CML: case study using baseline population characteristics

Kimbach Tran Carpiuc1, Gianantonio Rosti2, Fausto Castagnetti2, Maarten Treur3, Jennifer Stephens11Pharmerit North America LLC, Bethesda, MD, USA; 2Department of Hematology and Oncology, S Orsola-Malpighi University Hospital, Bologna, Italy; 3Pharmerit Europe, Rotterdam, The NetherlandsAbstract: The use of indirect comparisons to evaluate the relative effectiveness between two or more treatments is widespread in the literature and continues to grow each year. Appropriate methodologies will be essential for integrating data from various published clinical trials into a systematic framework as part of the increasing emphasis on comparative effectiveness research. This article provides a case study example for clinicians using the baseline study population characteristics and response rates of the tyrosine kinase inhibitors in imatinib-resistant or imatinib-intolerant chronic myelogenous leukemia followed by a discussion of indirect comparison methods that are being increasingly implemented to address challenges with these types of comparisons.Keywords: comparative effectiveness research, meta-analysis, BCR–ABL-positive chronic myelogenous leukemia, imatinib mesylate, nilotinib, dasatinib&nbsp