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Clinical outcomes in adult patients with aplastic anemia: A single institution experience.

ABSTRACT: Newer treatment modalities are being investigated to improve upon historical outcomes with standard immunosuppressive therapy (IST) in aplastic anemia (AA). We analyzed outcomes of adult patients with AA treated with various combinatorial anti-thymoglobulin-based IST regimens in frontline and relapsed/refractory (R/R) settings. Pretreatment and on-treatment clinical characteristics were analyzed for relationships to response and outcome. Among 126 patients reviewed, 95 were treatment-naïve (TN) and 63, R/R (including 32 from the TN cohort); median ages were 49 and 50 years, respectively. Overall survival (OS) was superior in IST responders (P?

SUBMITTER: Boddu P

PROVIDER: S-EPMC5987765 | biostudies-literature | 2017 Dec

REPOSITORIES: biostudies-literature

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Clinical outcomes in adult patients with aplastic anemia: A single institution experience.

Boddu Prajwal P Garcia-Manero Guillermo G Ravandi Farhad F Borthakur Gautam G Jabbour Elias E DiNardo Courtney C Jain Nitin N Daver Naval N Pemmaraju Naveen N Anderlini Paolo P Parmar Simrit S Kc Devendra D Akosile Mary M Pierce Sherry A SA Champlin Richard R Cortes Jorge J Kantarjian Hagop H Kadia Tapan T

American journal of hematology 20170925 12

Newer treatment modalities are being investigated to improve upon historical outcomes with standard immunosuppressive therapy (IST) in aplastic anemia (AA). We analyzed outcomes of adult patients with AA treated with various combinatorial anti-thymoglobulin-based IST regimens in frontline and relapsed/refractory (R/R) settings. Pretreatment and on-treatment clinical characteristics were analyzed for relationships to response and outcome. Among 126 patients reviewed, 95 were treatment-naïve (TN) ...[more]

PMID: 28850699

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Project description:Background: Rhabdomyosarcomas are the most common soft tissue sarcoma in children, and pediatric alveolar rhabdomyosarcoma (ARMS) prognosis has improved based on cooperative studies. However, in adults, ARMS is significantly rarer, has poorer outcomes, and currently lacks optimal treatment strategies. Objective: This study aimed to evaluate the clinical outcome of an adult ARMS population with different front-line systemic chemotherapies and determine if any chemotherapy regimen is associated with improved survival. Materials and methods: This is a retrospective study of histologically confirmed fusion-positive ARMS patients over 18 years of age, who were treated at MD Anderson Cancer Center (MDACC) from 2004 to 2021 and received systemic chemotherapy. Descriptive clinical statistics were performed, including staging, front-line chemotherapy, multimodal therapy usage, response rates, and survival analyses. Results: 49 ARMS patients who received upfront chemotherapy were identified. Locoregional treatments included radiotherapy (RT) alone (29%, n = 14), surgery alone (10%, n = 5), or both (45%, n = 22). Median overall survival (OS) for the entire cohort was 3.6 years, and the overall response rate to systemic therapy was 89%. No chemotherapy regimen showed OS benefit, specifically analyzing the pediatric-based vincristine, actinomycin-D, cyclophosphamide (VAC) or adult-based vincristine, doxorubicin, ifosfamide (VDI) regimens, even when controlled for other clinical risk factors. Conclusion: In this single-center contemporary series, adult ARMS patient outcomes remain poor. There was no statistically significant OS difference in patients who did or did not receive adult or pediatric based ARMS regimens, although a high overall response rate to chemotherapy was seen across the entire cohort. Based on these observations, further randomized prospective studies are necessary to delineate which frontline chemotherapy regimen is most beneficial in this rare adult cancer.

Project description:BackgroundDespite the increasing incidence of aplastic anemia in China, few studies have explored its effect on the patients' quality of life from the perspective of these patients. In fact, patients with aplastic disorder live with the disease for a long time, and need to face a variety of difficult realities, including multiple disease symptoms and drug side effects, heavy burden of medical costs, difficulties in social reintegration, and negative emotional distress. Therefore, this study used descriptive qualitative research to explore the direct and rich quality-of-life experiences of patients with aplastic anemia.MethodsA total of 19 patients with aplastic anemia were recruited in this study using purposive sampling combined with maximum variation strategy. 5 of the patients with AA were from northern China, and the others were from southern China. Data were collected using semi-structured interviews and analyzed using the conventional content analysis method.ResultsThis study yielded important information about the experiences of patients with aplastic anemia in China. The content analysis method finally identified 3 themes and 9 sub-themes, including: physical symptoms (declining physical capacity, treatment-related symptoms, changes in body image), psychological symptoms (mood changes related to the stage of the disease, change in self-image, growth resulting from the disease experience), social burden (decline in career development, perceived burden to the family, social stigma). Patients with AA from different regions didn't show much difference in quality of life.ConclusionsAplastic anemia affects the physical, psychological, and social aspects of patients' lives. Therefore, health care providers need to consider the patients' physical response and psychological feelings to provide relevant medical guidance and multi-channel social support that would improve their confidence and quality of life.Clinical trial registrationName: Development and preliminary application of Quality of Life Scale for Patients with Aplastic Anemia. Number: ChiCTR2100047575. URL: http://www.chictr.org.cn/login.aspx?referurl=%2flistbycreater.aspx .

Dataset Information

Clinical outcomes in adult patients with aplastic anemia: A single institution experience.

Publications

Clinical outcomes in adult patients with aplastic anemia: A single institution experience.

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