What Is the Impact on Quality of Life of Various Treatments in Acute Myeloid Leukemia and High-Risk Myelodysplastic Syndrome? Rethinking the Goals

Blood ◽  
2015 ◽  
Vol 126 (23) ◽  
pp. 2112-2112 ◽  
Author(s):  
Sara M. Tinsley ◽  
Brent J Small ◽  
Jeffrey E Lancet ◽  
Susan C McMillan ◽  
Rami S. Komrokji ◽  
...  

Abstract Introduction: Treatment decisions are difficult for older patients with acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS). Few studies address the impact of treatment on QOL. Both AML and high-risk MDS occur most frequently in the sixth and seventh decades of life, and are associated with a poor prognosis with median survival of one year or less. A primary goal of treatment is to improve quality of life (QOL) because cure is improbable. This was a longitudinal cohort study to compare QOL between groups receiving intensive therapy, non-intensive therapy, and supportive care. The sample consisted of 85 patients 60 years of age and older diagnosed high risk MDS and AML recruited from Moffitt Cancer Center from 12/2013 until 4/2015. Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) was used to measure QOL. Study aims were to: 1) To compare the difference in QOL scores measured by the Functional Assessment of Cancer Therapy-Leukemia version for intensive chemotherapy, non-intensive therapy and supportive care within 7 days of new treatment, or decision to pursue supportive care, and one month or later; 2) To determine QOL predictors of AML and high risk MDS from age, comorbidity, fatigue, and diagnosis; 3) To test the moderating effect of treatment with age, comorbidity, and fatigue on QOL. See figure 1. Methods: Recruitment of 85 patients with high risk MDS and AML occurred at the time of appointments in the Hematology Clinic or during admission to Moffitt Cancer Center for induction chemotherapy. Inclusion criteria included patients 60 years of age and older with confirmed diagnosis of high-risk MDS or AML based on bone marrow pathology report. High-risk MDS and AML were treatedas one group. Patients were able to read, write, and speak English, were orientedto person, place, and time, and werewilling to participate. Quality of life was assessedat the time of enrollment and within at least one month of enrollment using the FACT-Leu. Fatigue was measuredusing the Brief Fatigue Inventory, a one page, nine-item questionnaire, which measures fatigue on a scale of zero to ten, with zero indicating no fatigue, and ten representing the worst fatigue that a person can imagine. Measurement of number of comorbidities was performedat the time of enrollment using the Charlson comorbidity index. Baseline information obtained on all subjects included age, as measured by date of birth, and diagnosis from pathology report including chromosome analysis. Demographic data collected included gender, marital status, level of education, income level, religious ceremony attendance on a scale of zero to four, and designation of intensive, non-intensive, or supportive care treatment. Results: The first aim, a comparison of QOL scores from week 1 to week 4, was analyzed with repeated measures analysis of variance (ANOVA). The supportive care group was not included in the analysis because of low accrual. Results indicated that there was a significant group by time interaction (with p=.040). Follow up tests revealed that the intensive treatment group had a significant improvement in their QOL scores at 1 month post treatment (p=.020). The second aim, evaluation of predictors of QOL was conducted using Pearson's correlations with age, comorbidity, fatigue, and diagnosis with significant correlations found between fatigue and QOL (r=-.693, p< .001). These findings identify an important relationship between fatigue and QOL. This was a negative correlation, showing that as fatigue increased QOL decreased. The third aim was explored using regression with Hayes (2013) application for moderation analysis. Scores for QOL for age, comorbidity, and fatigue were not moderated by treatment. Conclusions: These findings suggest that the most intensive treatment approach improves QOL. In addition, fatigue is a significant predictor of QOL. As fatigue increased, QOL scores decreased. Additional studies with a larger, more diverse sample are needed to explore the relationship between treatment approaches and QOL. In addition, intervention studies can be developed in AML and high risk MDS focused on fatigue management. It is anticipated that the results of this study will be used to inform patients and health care providers when making decisions concerning treatment based on QOL outcomes. Figure 1. Figure 1. Figure 2. Figure 2. Disclosures Lancet: Seattle Genetics: Consultancy; Pfizer: Research Funding; Boehringer-Ingelheim: Consultancy; Kalo-Bios: Consultancy; Amgen: Consultancy; Celgene: Consultancy, Research Funding. Komrokji:Celgene: Consultancy, Research Funding; Incite: Consultancy; Novartis: Speakers Bureau; GSK: Research Funding. List:Celgene Corporation: Honoraria, Research Funding.

Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 4435-4435
Author(s):  
Jorge E. Cortes ◽  
Michael J. Mauro ◽  
Stuart L. Goldberg ◽  
Ron Paquette ◽  
H. Jean Khoury ◽  
...  

Abstract Abstract 4435 Background: Over the past decade, oral BCR-ABL inhibitors including imatinib, dasatinib and nilotinib have revolutionized chronic myeloid leukemia (CML) treatment. Clinical trials have provided evidence of improved outcomes with all three agents in the treatment of CML. However, limited information is available regarding the impact of disease and treatment on quality of life (QoL) in CML patients. Methods: SIMPLICITY is an ongoing 5-year USA/European prospective, observational cohort study (ClinicalTrials.gov ID: NCT01244750). Eligibility criteria include: newly diagnosed CML-CP, age ≥18 years and therapy with 1st-line imatinib, dasatinib or nilotinib at participating medical centers. The study aims to capture information on the use of these agents in clinical practice, including their impact on patient QoL. Through self-administered patient-reported outcomes (PRO) questionnaires, QoL and treatment-adherence data are being collected at 6-month intervals throughout study follow-up. Data from available initial PRO assessments of patients enrolled as of August 2011 are presented: Cancer Treatment Satisfaction Questionnaire (CTSQ), MD Anderson Symptom Inventory - CML (MDASI-CML), FACT-G (Functional Assessment of Cancer Therapy-General) and Morisky Medication Adherence Scale - 8 item (MMAS-8). Results: A total of 74 patients currently enrolled was included in this analysis of which 79.7% (n=59) received imatinib, 10.8% (n=8) received nilotinib and 9.5% (n=7) received dasatinib as 1st-line CML treatment. Median age was 57 years (range 19–94 years); a slightly higher proportion of patients were female (59.5%; n=44); and median time to completion of the PRO questionnaires from initiation of treatment was 383.5 days (range 4-1,225 days). Prior to completion of the PROs, mean treatment exposure was approximately 18 months for imatinib, 5 months for nilotinib and 4 months for dasatinib. Summary scores for the CTSQ, MDASI-CML and FACT-G are presented in Table 1. Due to small sample size, differences in scores between treatments were not statistically tested. At time of enrollment in the study, treatment satisfaction in this cohort was high (SWT score ± standard deviation [SD] =91.2 ±10.3). QoL, as measured by the FACT-G total and sub-scale scores, also indicated positive physical, social, emotional and functional well-being. Mean MDASI scores for Symptom Interference and Symptom Distress suggested the impact of symptoms was mild to moderate. From the MMAS-8, 71.8% (n=51) of patients reported medium/high adherence to their CML medication (scoring between 6–8 on an 8-point scale). Conclusions: Preliminary QoL evaluation suggests that newly diagnosed CML-CP patients are generally satisfied with treatment and report overall good health, although approximately 30% reported low adherence to their CML treatment. Initial assessment of the enrolled cohort suggests variance in treatment adherence patterns, as well as differences in the impact of symptoms by treatment; however, as these differences were not statistically tested, no firm conclusions can be drawn at this time. As patient enrollment continues, comparative effectiveness of these CML treatments and differences in QoL outcomes will be further observed. Disclosures: Cortes: Novartis: Research Funding; BMS: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Ariad: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Novartis: Consultancy. Mauro:BMS: Membership on an entity's Board of Directors or advisory committees, Research Funding; Novartis: Research Funding; Ariad: Research Funding. Goldberg:Novartis Pharmaceutical: Honoraria, Research Funding, Speakers Bureau; Bristol Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau. Paquette:BMS: Honoraria, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria. Khoury:BMS: Membership on an entity's Board of Directors or advisory committees. Hirji:BMS: Employment. Wagner:BMS: Employment. Joo:BMS: Employment. Davis:BMS: Employment.


2021 ◽  
pp. 107815522199844
Author(s):  
Abdullah M Alhammad ◽  
Nora Alkhudair ◽  
Rawan Alzaidi ◽  
Latifa S Almosabhi ◽  
Mohammad H Aljawadi

Introduction Chemotherapy-induced nausea and vomiting is a serious complication of cancer treatment that compromises patients’ quality of life and treatment adherence, which necessitates regular assessment. Therefore, there is a need to assess patient-reported nausea and vomiting using a validated scale among Arabic speaking cancer patient population. The objective of this study was to translate and validate the Functional Living Index-Emesis (FLIE) instrument in Arabic, a patient-reported outcome measure designed to assess the influence of chemotherapy-induced nausea and vomiting on patients’ quality of life. Methods Linguistic validation of an Arabic-language version was performed. The instrument was administered to cancer patients undergoing chemotherapy in a tertiary hospital's cancer center in Saudi Arabia. Results One-hundred cancer patients who received chemotherapy were enrolled. The participants’ mean age was 53.3 ± 14.9 years, and 50% were female. Half of the participants had a history of nausea and vomiting with previous chemotherapy. The Cronbach coefficient alpha for the FLIE was 0.9606 and 0.9736 for nausea and vomiting domains, respectively, which indicated an excellent reliability for the Arabic FLIE. The mean FLIE score was 110.9 ± 23.5, indicating no or minimal impact on daily life (NIDL). Conclusions The Arabic FLIE is a valid and reliable tool among the Arabic-speaking cancer population. Thus, the Arabic version of the FLIE will be a useful tool to assess the quality of life among Arabic speaking patients receiving chemotherapy. Additionally, the translated instrument will be a useful tool for future research studies to explore new antiemetic treatments among cancer patients.


2002 ◽  
Vol 20 (10) ◽  
pp. 2429-2440 ◽  
Author(s):  
Lewis R. Silverman ◽  
Erin P. Demakos ◽  
Bercedis L. Peterson ◽  
Alice B. Kornblith ◽  
Jimmie C. Holland ◽  
...  

PURPOSE: Patients with high-risk myelodysplastic syndrome (MDS) have high mortality from bone marrow failure or transformation to acute leukemia. Supportive care is standard therapy. We previously reported that azacitidine (Aza C) was active in patients with high-risk MDS.PATIENTS AND METHODS: A randomized controlled trial was undertaken in 191 patients with MDS to compare Aza C (75 mg/m2/d subcutaneously for 7 days every 28 days) with supportive care. MDS was defined by French-American-British criteria. New rigorous response criteria were applied. Both arms received transfusions and antibiotics as required. Patients in the supportive care arm whose disease worsened were permitted to cross over to Aza C.RESULTS: Responses occurred in 60% of patients on the Aza C arm (7% complete response, 16% partial response, 37% improved) compared with 5% (improved) receiving supportive care (P < .001). Median time to leukemic transformation or death was 21 months for Aza C versus 13 months for supportive care (P = .007). Transformation to acute myelogenous leukemia occurred as the first event in 15% of patients on the Aza C arm and in 38% receiving supportive care (P = .001). Eliminating the confounding effect of early cross-over to Aza C, a landmark analysis after 6 months showed median survival of an additional 18 months for Aza C and 11 months for supportive care (P = .03). Quality-of-life assessment found significant major advantages in physical function, symptoms, and psychological state for patients initially randomized to Aza C.CONCLUSION: Aza C treatment results in significantly higher response rates, improved quality of life, reduced risk of leukemic transformation, and improved survival compared with supportive care. Aza C provides a new treatment option that is superior to supportive care for patients with the MDS subtypes and specific entry criteria treated in this study.


Oncology ◽  
2014 ◽  
Vol 87 (3) ◽  
pp. 133-147 ◽  
Author(s):  
David Cella ◽  
Cindy J. Nowinski ◽  
Olga Frankfurt

Stroke ◽  
2013 ◽  
Vol 44 (suppl_1) ◽  
Author(s):  
Kristin J Addison-Brown ◽  
Abraham J Letter ◽  
Henry K Yaggi ◽  
Leslie A McClure ◽  
Frederick W Unverzagt ◽  
...  

Introduction: Using a subsample from the national REasons for Geographic And Racial Differences in Stroke (REGARDS) study, we examined the associations of obstructive sleep apnea (OSA) with cognition and quality of life and whether these associations vary with age while controlling for other demographic factors and comorbid medical conditions. Methods: Stroke-free participants with complete data on OSA risk, cognition, and quality of life as of October 2010 were included (N =2,925; ages 47-93, 43% men, 35% black, 65% white). OSA risk was defined as high or low based on responses to the Berlin Sleep Questionnaire (BSQ). Cognitive function was assessed with three validated fluency and recall measures; quality of life was assessed with the 4-item Center for Epidemiological Studies-Depression (CESD-4) scale and the Medical Outcomes Study Short Form-12 (SF-12). MANCOVA statistics were applied to the cognitive and quality of life outcomes separately while accounting for potential confounders (age, sex, race, education, diabetes and dyslipidemia). Body mass index and hypertension were taken into account as part of the BSQ definition of OSA risk. Results: In fully adjusted models, those at high risk for OSA had significantly lower cognitive scores (p < .05) and lower quality of life (depressive symptoms and SF-12) (p < .0001) than those at low risk. Some of the associations were age-dependent, such that younger participants with high OSA risk had worse cognitive and quality of life scores than both younger participants with low OSA risk and older participants with high OSA risk. Discussion: Lower cognitive function and lower quality of life in those at high risk for sleep apnea remained after accounting for potentially confounding factors in a population-based sample. These relationships were more pronounced during middle age, with attenuated effects after age 70. It may be of particular importance to detect and treat OSA in younger adults.


2020 ◽  
pp. bmjspcare-2020-002190
Author(s):  
Amanda Drury ◽  
Sheila Payne ◽  
Anne-Marie Brady

ObjectiveCancer survivors’ perceptions of healthcare have been identified as a predictor of quality of life (QoL) outcomes. This study aims to explore colorectal cancer (CRC) survivors’ perceptions of how cancer-related healthcare affects their QoL.MethodsSemistructured interviews were conducted with 22 CRC survivors receiving follow-up care between 1 and 5 years post diagnosis. Interviews were recorded, transcribed and analysed thematically.ResultsFive themes described the impact of healthcare experiences on CRC survivors’ QoL. While cancer survivors spoke positively of their relationships with healthcare professionals, many experienced a range of unmet information and supportive care needs. Participants described a range of positive and negative experiences, as power dynamics and navigation of healthcare systems had implications for their QoL. Where negative healthcare events aligned, survivors’ autonomy, dignity and confidence were undermined, and survivorship issues could be inadequately addressed. To address persistent unmet needs, survivors developed a safety net(work) of supports to bridge the gap of unmet needs in healthcare with varying outcomes.ConclusionsCancer survivors’ experience of follow-up and healthcare can positively or negatively affect their QoL. Preparation for cancer survivorship must be incorporated into the acute phase of diagnosis and treatment and interlinked with clear pathways of survivorship care and accessible supportive care, which support survivors to be equal partners in their healthcare. Understanding cancer survivors’ knowledge, expertise and mastery of their condition is essential to ensure delivery of person-centred supportive care that adequately addresses the survivor’s unmet needs.


2018 ◽  
Vol 4 (Supplement 2) ◽  
pp. 104s-104s
Author(s):  
V.S. Mishra ◽  
S. Chandrakala ◽  
D. Saranath

Background: Studies have demonstrated poor quality of life (QOL) among cancer caregivers, but few studies have evaluated QOL and related psychosocial functioning among caregivers for people with hematologic cancer patients. The disease and its associated treatment can have overwhelming consequences for patients and their informal caregivers mostly close family members. Aim: The aim of the study was to analyze the impact of cancer diagnosis and its treatment in caregivers´ quality of life (QoL) and to observe the association with perceived social support. To compare the chronic myeloid leukemia patients to other leukemia patients caregiver to find differences and understand which aspects of QoL are more impacted. Methods: The current study is on-going study; sample is composed of caregivers of patients with chronic myeloid leukemia patients and acute myeloid leukemia, acute lymphoblastic leukemia and other patient's caregiver from the Department of Hematology, KEM Hospital, Mumbai India. We are using Caregiver Quality of Life Index-Cancer (CQOLC) for measuring quality of life among caregivers of cancer patients and Multidimensional Scale of Perceived Social Support (MSPSS) for social support. Results: With the small study sample of caregivers of patients mean age 34.78 ± 6.87 with the age range of 27-45 years, 25% of whom were men. Mean QOL score is 78.34 ± 15.53. In regression analysis caregivers education has shown significant t = 5.611, < 0.05. While marital status shown significant value with social support 3.931, 3.872, < 0.01. While CML caregivers had lower scores for the quality of life and less perceived social support. Conclusion: Caregivers of patients with cancer showed increased burden scores and financial issues and less perceived social support. This could be explained by their unique care situation. These caregivers need more attention and additional counseling session for coping with the situation.


2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 6537-6537
Author(s):  
Brooke Worster ◽  
Valerie P Csik ◽  
Jared Minetola ◽  
Gregory D. Garber ◽  
Alison Petok ◽  
...  

6537 Background: Evidence suggests that cancer patients who receive palliative care early in their disease have improved quality of life, decreased emergency department (ED) visits, and less aggressive end-of-life care. In 2017, the Sidney Kimmel Cancer Center at Jefferson established the Neu Center for Supportive Medicine and Cancer Survivorship (NCSMCS) as a model for integrated care in the outpatient setting for all cancer patients. A multidisciplinary team consisting of palliative care physicians, social work, psychology, and navigation conducts biopsychosocial screening and initiates a personalized care plan for each patient to clarify treatment goals and offer assistance. Objectives: To use biopsychosocial screening at specified time points to identify needs and evaluate the impact of supportive care as part of standardized oncology care regardless of stage. Methods: This assessment utilized Oncology Care Model (OCM) data for Jefferson Medicare patients between 7/1/16 to 7/31/18. Incidence of ED admits ED/Observation and admissions were evaluated as well as ICU utilization and advanced care planning. Poisson regression was used to generate incidence rate ratios (IRR) and 95% confidence intervals (CI) to facilitate the comparison of post- vs. pre- incidence rates of hospitalization. Results: The post-intervention hospital admissions decreased by 31% in NCSMCS (IRR 0.69; 95% CI 0.48-0.98) and by 10% in Non-NCSMCS (IRR 0.90; 0.84-0.96) and advanced care plans were more likely to be on file for NCSMCS (9.0% vs. 4.9%). The intensive care unit (ICU) admissions were decreased by 17% among Non-NCSMCS (IRR 0.83; 95% CI 0.74-0.93). The utilization rates for ED admissions were not statistically different among both the groups. Conclusions: The preliminary data is promising and impact will be monitored as the intervention is expanded. Reducing admissions has benefits from both a cost savings as well as quality of life perspective. Future analyses will consider the impact of the intervention on a patient’s quality of life.


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 5049-5049 ◽  
Author(s):  
Stephen P McKenna ◽  
James Twiss ◽  
Jeanette N Wilburn ◽  
Sigrid Rebecca Crawford ◽  
Kevin Loth ◽  
...  

Abstract Abstract 5049 Introduction: Little research is currently available on the impact of MDS from the patients' perspective. Where research has been conducted it has predominantly used generic cancer outcome measures. Such measures do not provide an accurate assessment of MDS patients as they miss important aspects of the condition. A qualitative study was conducted with MDS patients to investigate how their lives are affected by the condition. Methods: Qualitative, unstructured interviews were conducted with patients. Verbatim transcripts of the interviews were analysed thematically to assess the impact of MDS in terms of symptoms experienced, activity limitations and quality of life (QoL). Emergent themes were identified, clustered and harmonised. Investigation of issues related to impairments and activity limitations were guided by the World Health Organizations (WHO) classification of outcomes related to health and injury. Investigation of QoL issues was guided by the needs-based model of QoL (Hunt SM, McKenna SP. The QLDS: A scale for the measurement of quality of life in depression. (1992). Health Policy 22; 307–319). This model identifies several core fundamental needs that are important to an individual's QoL and adjustment to society. Illness affects QoL by preventing the satisfaction of these needs. Results: The sample included 30 participants (male 17 (56.7%); mean (SD) age 65.5 (11.3) years with low to intermediate-1 risk MDS. Illness information for the sample is shown in Table 1. The analyses identified several symptoms. The most frequently reported symptoms were; fatigue (97%), breathlessness (60%), increased infections (60%), sleep problems (53%), cognitive problems (50%), temperature fluctuations (47%), increased bruising (40%) and bleeding (10%), pain (30%), depression (23%) and anxiety (10%). Several common functions were affected by MDS, including; standing, walking, bending, lifting, carrying and rising from sitting. Various more complex functions were also affected such as the ability to socialise, shop and do jobs around the house. A total 33 QoL issues were identified. Nine fundamental needs were affected by MDS; subsistence, safety and security, affection, understanding, participation, leisure, self-actualisation, identity and independence. Conclusions: The study was successful in developing outcome models describing how MDS affects symptoms, activity limitations and QoL. Many symptoms were experienced including several in addition to those directly associated with anaemia. Functioning was also frequently affected. A wide range of QoL issues were identified with several fundamental needs impaired to some extent. Following this study a large bank of potential items has been created that will aid the development of MDS-specific outcome scales assessing symptoms, activity limitations and QoL. Disclosures: McKenna: Celgene Ltd: Consultancy, Research Funding. Twiss:Celgege Ltd: Consultancy, Research Funding. Wilburn:Celegene Ltd: Consultancy, Research Funding. Crawford:Celegene: Consultancy, Research Funding. Loth:Celgene Ltd: Employment.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 3472-3472 ◽  
Author(s):  
Anita Hill ◽  
Richard J Kelly ◽  
Austin G Kulasekararaj ◽  
Shreyans A Gandhi ◽  
Lindsay D Mitchell ◽  
...  

Abstract Abstract 3472 Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal stem cell disorder arising on the background of bone marrow failure and resulting in hemolytic anemia, thrombosis, pulmonary hypertension (PHT) and chronic kidney disease (CKD) through uncontrolled complement activation. Approximately half of patients treated with supportive therapies alone die as a result of their PNH. Eculizumab blocks C5 and thereby terminal complement activation. Complications are therefore prevented with reduction in intravascular hemolysis, transfusion requirements, thromboses, pulmonary pressures and renal impairment all associated with significantly improved quality of life. The UK has a nationally commissioned PNH service led by 2 Centres, Leeds and King's. Between May 2002 - April 2012, a total of 153 patients (76 male; 77 female) were treated with eculizumab for PNH in the UK for a mean of 42 mths (0.4 – 119). Patients are treated if they have 1) transfusion-dependent hemolysis or 2) independent of transfusions, either i) thrombosis, ii) CKD, iii) PHT, iv) pregnancy or v) LDH&gt;1.5× upper limit normal with anemia and symptoms including fatigue, dysphagia, dyspnea or abdominal pain due to PNH. Median age at PNH diagnosis was 34 yrs (range 12–80) and at initiation of eculizumab was 42 yrs (range 14–84). There were 28 thrombotic episodes in 15/65 (23%) patients anticoagulated prior to commencing eculizumab therapy. Nine patients have died - 3 due to progression of their underlying bone marrow failure to MDS/AML, one died immediately after BMT and the remaining 5 deaths are not directly related to PNH. Seven patients discontinued eculizumab - one had predominant aplastic anemia (AA), 2 had spontaneous remissions of the PNH clone, 3 commenced for a pregnancy discontinuing after and one who had a successful transplant for very severe AA. One hundred and thirty-seven patients remain on eculizumab. There were 36 thrombotic episodes in 22 patients in the 12 months prior to eculizumab and 3 thromboses in 3 patients in the most recent 12 months on therapy (1 Budd-Chiari during complement blockade breakthrough caused by infection, 1 CVA during reversal of coumadin overanticoagulation, 1 TIA/lacunar infarct thought to be due to diabetic small vessel disease) (P&lt;0.05). Of the 22 patients who had a thrombosis within 12 months prior to starting eculizumab there were no further thrombotic episodes once on eculizumab. Importantly, primary and secondary prophylaxis with warfarin has been safely stopped in 43 and 4 patients respectively, with no thrombotic sequelae. Ten patients had not required a transfusion before receiving eculizumab. Of the 117 patients transfused in the 12 months before receiving eculizumab and on therapy for the most recent 12 months, 77 (66%) became transfusion independent. Of the 40 patients still needing transfusions, there was a significant (P&lt;0.05) reduction in the number of units transfused from a median of 26 units 12 months before therapy to 8 units in the most recent 12 months on therapy. There were 3 cases of meningococcal septicaemia out of 153 patients treated (0.6 cases/100 patient yrs on therapy) with none in the last 2 yrs since routine penicillin prophylaxis was instituted. All were promptly and effectively managed and remain on eculizumab therapy. The survival of UK PNH patients on eculizumab was compared with age and sex matched controls (Fig 1) and demonstrates improved survival compared with historical controls. Survival after 10 years is slightly inferior to controls with causes of death related to bone marrow failure and not hemolysis or thrombosis. Conclusions: the total UK cohort provide more than 10 years experience of eculizumab for PNH and shows a) continuing safety and efficacy of eculizumab with persistent significant improvement in symptoms and quality of life b) no evidence of tolerance or refractoriness c) significant reduction in thrombosis risk on eculizumab therapy and safety in discontinuing primary anticoagulation d) significant reduction in the need for transfusions e) progression to MDS/AML as expected for the underlying bone marrow failure and not considered related to eculizumab and f) significant improvement in survival for PNH patients receiving eculizumab. This cohort of all PNH patients treated with eculizumab in the UK demonstrates the impact of eculizumab in the quality of life, reduction in complications and thereby improved survival for patients. Disclosures: Hill: Alexion Pharmaceuticals, Inc.: Consultancy, Honoraria. Kelly:Alexion Pharmaceuticals, Inc.: Consultancy, Honoraria. Gandhi:Alexion Pharmaceuticals, Inc.: Research Funding. Mitchell:Alexion Pharmaceuticals, Inc: Honoraria. Elebute:Alexion Pharmaceuticals, Inc.: Consultancy, Honoraria, Research Funding. Arnold:Alexion Pharmaceuticals, Inc.: Honoraria. Marsh:Alexion Pharmaceuticals, Inc.: Honoraria. Hillmen:Alexion Pharmaceuticals, Inc: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees.


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