Eculizumab in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Report of All 153 Patients Treated in the UK

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>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<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<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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Attitudes About COVID-19 and Health: A Mixed-Methods Smartphone-App and Online Survey Study (Preprint)

10.2196/preprints.29963 ◽

2021 ◽

Author(s):

Anna M. Hood ◽

Hanne Stotesbury ◽

Jennifer Murphy ◽

Melanie Kölbel ◽

April Slee ◽

...

Keyword(s):

Quality Of Life ◽

Mental Health ◽

Data Collection ◽

Physical Health ◽

Online Survey ◽

Smartphone App ◽

The Uk ◽

The Impact ◽

Monthly Survey

BACKGROUND Behavioral mitigation strategies to slow the spread of COVID-19 have resulted in sweeping lifestyle changes, with short and long-term psychological, well-being, and quality of life implications. The Attitudes About COVID-19 and Health (ATTACH) study focuses on understanding attitudes and beliefs whilst considering the impact on mental and physical health and the influence of broader demographic and geographic factors on attitudes, beliefs, and mental health burden. OBJECTIVE In this assessment of our first wave of data collection, we provide baseline cohort descriptives of ATTACH study participants in the United Kingdom (UK), United States of America (USA), and Mexico. Additionally, we assess responses to daily poll questions related to COVID-19 and conduct a cross-sectional analysis of baseline assessments collected in the UK between June 26 and October 31, 2020. METHODS The ATTACH study uses smartphone-app technology and online survey data collection. Participants completed poll questions twice daily related to COVID-19 and a monthly survey assessing mental health, social isolation, physical health, and quality of life. Poll question responses were graphed using 95% Clopper-Pearson (exact) tests with 95% confidence intervals. Pearson correlations, hierarchical linear regression analyses, and generalized linear models assessed relationships, predictors of self-reported outcomes, and group differences, respectively. RESULTS By October 31, 2020, 1405, 80, and 90 participants had consented to participate in the UK, USA, and Mexico, respectively. Descriptive data for the UK daily poll questions indicated that participants were generally following social distancing measures, but worry and negative impacts on families increased as the pandemic progressed. Although participants generally reported feeling that the reasons for current measures had been made clear, there was low trust that the government was doing everything in its power to meet public needs. In the UK, 1282 participants also completed a monthly survey (95% white, 72% female, 21% key or essential workers). Nineteen percent of UK participants reported a pre-existing mental health disorder, 31% reported a pre-existing chronic medical illness, and 35% were over 65. Fifty-seven percent of participants reported being more sedentary since the pandemic began, and 41% reported reduced access to medical care. Those with poorer mental health outcomes lived in more deprived neighborhoods, in larger households (ps < .05), had more pre-existing mental health disorders and medical conditions, and were younger than 65 years (all ps < .001). CONCLUSIONS Communities who have been exposed to additional harm during the COVID-19 pandemic were experiencing worse mental outcomes. Factors including having a medical condition, or living in a deprived neighborhood or larger household were associated with heightened risk. Future longitudinal studies should investigate the link between COVID-19 exposure, mental health, and sociodemographic and residential characteristics.

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Validation of the English Version of the Scale for Psychosocial Factors in Food Allergy and the Relationship with Mental Health, Quality of Life, and Self-Efficacy

Journal of Allergy ◽

10.1155/2016/4850940 ◽

2016 ◽

Vol 2016 ◽

pp. 1-7 ◽

Cited By ~ 3

Author(s):

Rebecca C. Knibb ◽

Aaron Cortes ◽

Christopher Barnes ◽

Carol Stalker

Keyword(s):

Quality Of Life ◽

Mental Health ◽

Food Allergy ◽

Psychosocial Factors ◽

Self Efficacy ◽

Holistic View ◽

Parental Quality ◽

The Uk ◽

The Impact

Background. The Scale for Psychosocial Factors in Food Allergy (SPS-FA) is based on the biopsychosocial model of health and was developed and validated in Chile to measure the interaction between psychological variables and allergy symptoms in the child. We sought to validate this scale in an English speaking population and explore its relationship with parental quality of life, self-efficacy, and mental health. Methods. Parents (n=434) from the general population in the UK, who had a child with a clinical diagnosis of food allergy, completed the SPS-FA and validated scales on food allergy specific parental quality of life (QoL), parental self-efficacy, and general mental health. Findings. The SPS-FA had good internal consistency (alphas = .61–.86). Higher scores on the SPS-FA significantly correlated with poorer parental QoL, self-efficacy, and mental health. All predictors explained 57% of the variance in SPS-FA scores with QoL as the biggest predictor (β=.52). Discussion. The SPS-FA is a valid scale for use in the UK and provides a holistic view of the impact of food allergy on the family. In conjunction with health-related QoL measures, it can be used by health care practitioners to target care for patients and evaluate psychological interventions for improvement of food allergy management.

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The patient’s experience of primary ciliary dyskinesia: a systematic review

Quality of Life Research ◽

10.1007/s11136-017-1564-y ◽

2017 ◽

Vol 26 (9) ◽

pp. 2265-2285 ◽

Cited By ~ 10

Author(s):

Laura Behan ◽

Bruna Rubbo ◽

Jane S. Lucas ◽

Audrey Dunn Galvin

Keyword(s):

Quality Of Life ◽

Primary Ciliary Dyskinesia ◽

Age Groups ◽

Health Related Quality ◽

Related Quality ◽

Health Related ◽

The Uk ◽

The Impact ◽

Ciliary Dyskinesia

Abstract Background Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by progressive sinopulmonary disease, with symptoms starting soon after birth. The aim of this study is to critically review, analyse, and synthesise the literature in order to understand the experiences of patients with primary ciliary dyskinesia (PCD) and the impact on health-related quality of life. Method MEDLINE, EBSCO, Cumulative Index to Nursing and Allied Health Literature (CINAHL), PsycINFO and EMBASE were searched according to the inclusion criteria. A qualitative analysis of 14 studies was conducted. Results Fourteen studies were included in the review, five with qualitative methodologies. Studies originated from the UK, USA, Italy, Denmark and Belgium, one study included a survey distributed internationally. Significant relationships were found between age and worsening of respiratory symptoms, physical, and mental domains of health-related quality of life, with a greater decline compared with reference populations. Variations between the UK and Italy were found for health-related quality of life and its correlation with time since diagnosis. PCD was found to have a physical impact in all age groups: patients found it difficult to keep up with others, and found energy levels were easily depleted compared to family or peers. In terms of social impact, symptoms lead to embarrassment and a sense of isolation, with patients concealing symptoms and/or their diagnosis. In turn, isolation was also linked with the lack of public and medical knowledge. In relation to emotional impact, anxiety was reported in a number of qualitative studies; patients were anxious about getting sick or when thinking about their future health. The burden of treatment and factors influencing adherence were also discussed in depth. Conclusion Health-related quality of life decreases with age in patients with PCD. For all age groups, PCD was found to greatly impact physical, emotional, social functioning, and treatment burden. More research is needed on the psychosocial impact of the illness, disease burden and its effect on quality of life.

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952 THE IMPACT OF PRIMARY BILIARY CIRRHOSIS (PBC) ON PERCEIVED QUALITY OF LIFE (QoL): THE UK-PBC NATIONAL STUDY

Journal of Hepatology ◽

10.1016/s0168-8278(13)60954-x ◽

2013 ◽

Vol 58 ◽

pp. S392

Author(s):

G. Mells ◽

G. Pells ◽

J. Newton ◽

A. Bathgate ◽

A. Burroughs ◽

...

Keyword(s):

Quality Of Life ◽

Primary Biliary Cirrhosis ◽

Perceived Quality ◽

National Study ◽

Biliary Cirrhosis ◽

Perceived Quality Of Life ◽

The Uk ◽

The Impact

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Impact of therapy with epoetin alfa on clinical outcomes in patients with nonmyeloid malignancies during cancer chemotherapy in community oncology practice. Procrit Study Group.

Journal of Clinical Oncology ◽

10.1200/jco.1997.15.3.1218 ◽

1997 ◽

Vol 15 (3) ◽

pp. 1218-1234 ◽

Cited By ~ 522

Author(s):

J Glaspy ◽

R Bukowski ◽

D Steinberg ◽

C Taylor ◽

S Tchekmedyian ◽

...

Keyword(s):

Quality Of Life ◽

Functional Status ◽

Cancer Patients ◽

Hemoglobin Level ◽

Activity Level ◽

Epoetin Alfa ◽

Open Label ◽

Transfusion Requirements ◽

The Impact

PURPOSE To study the impact of Procrit (epoetin alfa; Amgen Inc, Thousand Oaks, CA) on quality of life, transfusion requirements, and hemoglobin in anemic cancer patients receiving chemotherapy. PATIENTS AND METHODS More than 500 community-based oncologists enrolled 2,342 patients with malignancies undergoing cytotoxic chemotherapy in an open-label study. Patients were treated with epoetin alfa 150 U/kg three times weekly, which could be doubled if the therapuetic response was judged inadequate. Total treatment was up to 4 months. RESULTS Of the 2,342 patients enrolled, data were available for 2,030 patients. Of the 2,030, 1,047 patients completed all 4 months of epoetin alfa therapy. Epoetin alfa was associated with significant increases in mean self-rated scores for energy level, activity level, and overall quality of life; these improvements correlated with the magnitude of the hemoglobin increase and were independent of tumor response. In addition, epoetin alfa was associated with a significant increase in mean hemoglobin and with a significant decrease in the proportion of patients requiring transfusions (baseline to final value, P < .001). Epoetin alfa was well tolerated. CONCLUSION Epoetin alfa is effective in improving the functional status and quality of life in anemic cancer patients receiving chemotherapy, as well as increasing hemoglobin level and decreasing transfusion requirements. Improvement in functional status can be attributed to an increase in hemoglobin level, demonstrating that quality of life in this group of patients can be improved by aggressively treating anemia. Further studies will be required to define the optimal doses and schedules for epoetin alfa.

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Disease-Related Symptoms Reported across a Broad Population of Patients with Paroxysmal Nocturnal Hemoglobinuria.

Blood ◽

10.1182/blood.v110.11.3683.3683 ◽

2007 ◽

Vol 110 (11) ◽

pp. 3683-3683 ◽

Cited By ~ 8

Author(s):

Gabrielle Meyers ◽

Ilene Weitz ◽

Thierry Lamy ◽

Jean-Yves Cahn ◽

Henk-André Kroon ◽

...

Keyword(s):

Quality Of Life ◽

Erectile Dysfunction ◽

Paroxysmal Nocturnal Hemoglobinuria ◽

Blood Clot ◽

Signs And Symptoms ◽

Poor Quality ◽

Shortness Of Breath ◽

European Organization ◽

Transfusion Requirements

Abstract In paroxysmal nocturnal hemoglobinuria (PNH), lack of the GPI-anchored terminal complement inhibitor CD59 from erythrocytes renders them susceptible to chronic hemolysis, which is central to the signs and symptoms of PNH. Patients are at elevated risk for thrombosis, experience anemia that may require transfusion support, and suffer from fatigue that can be severe. Patients often have a poor quality of life resulting from PNH related symptoms including pain, dyspnea, dysphagia and erectile dysfunction, which negatively impact quality of life. The prevalence and severity of symptoms were explored in the context of a multi-national content validation study, of patients not receiving eculizumab therapy, employing the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue and the European Organization for Research and Treatment of Cancer Quality of Life Core 30 (EORTC QLQ-C30) instruments. Symptom questions were asked of 29 PNH patients (19 men, 10 women, mean age 41.2±13.2 years) from the United Kingdom, United States, France and Spain. More than half (52%) had PNH for over 5 years. Most (76%) reported never having had a blood clot, 31% reported not receiving any medication for their PNH, and 59% reported either that they had never been transfused or had not received transfusion within the last year for PNH. Patients viewed overall quality of life, global health, functioning, fatigue, pain, and shortness of breath as important PNH-related signs/symptoms. Both the FACIT-Fatigue and EORTC instruments were relevant and adequate in assessing the level of fatigue and other quality of life measures in PNH. The burden of disease in this multicultural and diverse cohort of patients was significant: 76% were forced to modify their daily activities to manage their PNH and 17% were unemployed due to PNH. Nearly all (96%) complained of fatigue and more than half reported abdominal pain, headache and shortness of breath (Table). Patients also commonly reported dysphagia (41%) and erectile dysfunction (47% in males). Most patients reported these PNH-related symptoms as moderate to very severe, and a substantial majority reported distress associated with the symptoms. Significant disease burden was identified in a diverse population of PNH patients, most of which had minimal or no transfusion requirements and a low incidence of thrombosis. Therapy that controls hemolysis and thereby improves fatigue, pain, shortness of breath, dysphagia and erectile dysfunction may prove beneficial for PNH patients with these disease characteristics.

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Home Infusion of Eculizumab: A Unique and Innovative Model of Drug Delivery to Reduce Treatment-Associated Burden and Enhance Quality of Life for Patients with PNH

Blood ◽

10.1182/blood.v112.11.4671.4671 ◽

2008 ◽

Vol 112 (11) ◽

pp. 4671-4671

Author(s):

Louise M Arnold ◽

Jill Stephenson ◽

Richard Kelly ◽

David Buchanan ◽

Gareth Jones ◽

...

Keyword(s):

Quality Of Life ◽

Bone Marrow ◽

Financial Burden ◽

Well Being ◽

Full Time ◽

Home Infusion ◽

The Uk ◽

Full Time Employment ◽

At Home

Abstract Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal stem cell disease, characterised by intravascular hemolysis, bone marrow failure and lifethreatening thromboses. The median survival is 10–15 years, with the average age of presentation being in the 30’s. Symptoms include hemoglobinuria, fatigue, anemia, venous and arterial thromboses, recurrent pain, renal impairment, erectile dysfunction and pulmonary hypertension. The care of a patient with PNH is complex and challenging, as many experience chronic symptoms with periods of acute exacerbations. Historically the management of PNH included bone marrow transplant, blood transfusion and administration of additional supportive therapies, all necessitating regular visits to the hospital. Eculizumab, a monoclonal antibody that binds to the C5 complement component inhibiting the activity of terminal complement and thus preventing the destruction of red blood cells has dramatically altered the management of hemolytic PNH. Clinical trials of eculizumab demonstrated the resolution of the majority of symptoms and complications of PNH and resulted in its approval in the UK in June 2007. Eculizumab is administered as a 30 minute intravenous infusion every 14 days, and under the terms of its current EU licence, must be administered by a healthcare professional. In view of the rarity of PNH there are relatively few specialist Centres for the disease resulting in, patients travelling long distances for review and treatment. In view of the dramatic improvement in symptoms on eculizumab many patients are able to return to a near normal lifestyle. In the UK, Leeds Teaching Hospitals with Healthcare at Home have developed a home infusion programme that ensures safe administration of eculizumab in the patient’s home at a time convenient to them, leading to enhanced treatment-associated convenience for patients and their families. Patients then only attend the PNH Centre every 3 months to ensure appropriate monitoring and patient education. A recent survey of patients reports a reduction in treatment-associated burden for PNH patients and their families when receiving infusions at home. 46 patients responded to the survey with just over half receiving eculizumab. Of the 21 patients at the time receiving home infusions 19 found this more convenient than the hospital. Home treatment allows flexibility and for some, the return to full-time employment, with the associated financial benefits and improvement in psychological well-being. Of the 21 patients on home care 7 stated there ability to work was transformed with a further 10 having great improvement. Whilst the purpose of the survey was not to address financial burden, the home infusion programme has anecdotally reduced the financial burden on the patient and their family by eliminating the need for time off work, allowing return to full-time employment, and eliminating the cost of travel to and from the hospital for treatments. No patients reporting negative impact, including effect on social life and family relationships, whilst 15 experienced improvement or complete transformation in both areas. The patients reported confidence in the homecare programme, knowing that a very close working relationship existed between the expert hospital and homecare teams. This innovative programme of medication delivery by a dedicated home nursing team allows patients who have previously struggled to cope with their illness to lead a near normal life with an associated enhancement in quality of life. Patients are able to carry on with activities of daily life, including work, recreational activities and holidays, whilst at the same time ensuring compliance with treatment and therefore allowing maximum therapeutic benefit.

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

Blood ◽

10.1182/blood.v126.23.2112.2112 ◽

2015 ◽

Vol 126 (23) ◽

pp. 2112-2112 ◽

Cited By ~ 1

Author(s):

Sara M. Tinsley ◽

Brent J Small ◽

Jeffrey E Lancet ◽

Susan C McMillan ◽

Rami S. Komrokji ◽

...

Keyword(s):

Quality Of Life ◽

High Risk ◽

Supportive Care ◽

Myeloid Leukemia ◽

Research Funding ◽

Intensive Therapy ◽

Cancer Center ◽

Pathology Report ◽

The Impact

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.

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The impact of a dedicated physiotherapist clinic for children with dysfunctional breathing

ERJ Open Research ◽

10.1183/23120541.00103-2015 ◽

2016 ◽

Vol 2 (3) ◽

pp. 00103-2015 ◽

Cited By ~ 8

Author(s):

Nicola J. Barker ◽

Heather Elphick ◽

Mark L. Everard

Keyword(s):

Quality Of Life ◽

Long Term Effects ◽

Time Points ◽

Significant Difference ◽

Breathing Retraining ◽

Dysfunctional Breathing ◽

The Uk ◽

The Impact ◽

Time Point

Dysfunctional breathing is a significant cause of morbidity, adversely affecting an individual's quality of life. There is currently no data from paediatric centres on the impact of breathing retraining for dysfunctional breathing.Symptoms and quality of life were measured in 34 subjects referred sequentially for breathing retraining to the first dedicated paediatric dysfunctional breathing clinic in the UK. Data were obtained prior to the first intervention (time point 1), at discharge (time point 2) and by post 6 months later (time point 3).The mean (interquartile range) age of participants was 13.3 (9.1–16.3) years, with 52% female. Data were obtained at time points 2 and 3 in 23 and 13 subjects, respectively.Statistically significant improvements were observed in symptom scores, child quality of life and parental proxy quality of life between time points 1 and 2 (p<0.0001), while there was no significant difference in the data at time point 3 as compared with time point 2.This study suggests that physiotherapist-led breathing retraining offers significant benefit to young people with dysfunctional breathing which is maintained for at least 6 months after treatment is completed. Future studies will provide more information on the long-term effects of interventions for dysfunctional breathing.

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