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QUALITY OF LIFE AND SYMPTOM BURDEN IN MYELOFIBROSIS PATIENTS:A REAL WORLD STUDY
Author(s): ,
Tatiana Ionova
Affiliations:
Multinational Center for Quality of Life Research,Saint-Petersburg,Russian Federation;Saint-Petersburg MultiSpeciality Medical Center,Saint-Petersburg,Russian Federation
,
Lyudmila Anchukova
Affiliations:
Vologda Regional Clinical Hospital,Vologda,Russian Federation
,
Olga Vinogradova
Affiliations:
Hematological City Center,Botkin City Clinical Hospital,Moscow,Russian Federation;Pirogov Russian National Research Medical University,Moscow,Russian Federation
,
Taras Gritsenko
Affiliations:
Samara State Medical University,Samara,Russian Federation
,
Andrey Evseev
Affiliations:
Republican Hospital named after V.A. Baranov,Petrozavodsk,Russian Federation
,
Liliya Kozlova
Affiliations:
Orenburg State Medical University,Orenburg,Russian Federation
,
Kira Kurbatova
Affiliations:
Multinational Center for Quality of Life Research,Saint-Petersburg,Russian Federation;Saint-Petersburg MultiSpeciality Medical Center,Saint-Petersburg,Russian Federation
,
Galina Kuchma
Affiliations:
Orenburg State Medical University,Orenburg,Russian Federation
,
Elza Lomaia
Affiliations:
Federal Almazov North-West Medical Research Center,Saint-Petersburg,Russian Federation
,
Anna Lyamkina
Affiliations:
Novosibirsk State Medical University,Novosibirsk,Russian Federation
,
Elena Machulaytene
Affiliations:
Outpatient diagnostic department,Pavlov State Medical University,Saint-Petersburg,Russian Federation
,
Alexander Myasnikov
Affiliations:
Republican Hospital named after V.A. Baranov,Petrozavodsk,Russian Federation
,
Tatiana Nikitina
Affiliations:
Multinational Center for Quality of Life Research,Saint-Petersburg,Russian Federation;Saint-Petersburg MultiSpeciality Medical Center,Saint-Petersburg,Russian Federation
,
Maria Pankrashkina
Affiliations:
Hematological City Center,Botkin City Clinical Hospital,Moscow,Russian Federation
,
Tatiana Pospelova
Affiliations:
Novosibirsk State Medical University,Novosibirsk,Russian Federation
,
Anna Rodionova
Affiliations:
Multinational Center for Quality of Life Research,Saint-Petersburg,Russian Federation
Elena Usacheva
Affiliations:
R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation,Pavlov State Medical University,Saint-Petersburg,Russian Federation
(Abstract release date: 05/19/16) EHA Library. Ionova T. 06/09/16; 134925; PB2025
Prof. Tatyana Ionova
Prof. Tatyana Ionova
Contributions
Abstract
Abstract: PB2025

Type: Publication Only

Background
Myelofibrosis (MF) is associated with poor prognosis, significant symptom burden, and worsened quality of life (QoL). Assessment of patient-reported outcomes is an effective way to identify patients’ risks/benefits of MF treatment. 

Aims
We aimed to study QoL and symptom burden in MF pts in a real world setting as well as to make comparisons in the groups depending on treatment modality.

Methods
A total 93 pts with MF were enrolled in the multicenter real world QoL study – 64 (69%) with primary MF, 14 (15%) post-essential thrombocytopenia MF, 15 (16%) post-polycythemia Vera MF. Median age – 59 years (range 28–90); male/female – 37/56. All pts received the best available treatment (BAT, n=62) or ruxolitinib (n=31) for at least 6 months (median 12 months, range 6–238). High proportion of pts (79%) had IPSS intermediate or high risk. All pts completed the SF-36 QoL questionnaire, symptom assessment questionnaire CSP-MF and Patient Global Impression of Change (PGIC) tool; physicians collected information about disease and treatment from medical records. Integral QoL Index was calculated on the basis of SF-36 QoL questionnaire. For statistical analysis ANOVA, multiple regression and χ2 test were applied.

Results
23 pts (24.7%) from 93 pts were full spleen reduction responders, 31 pts (33.3%) – moderate responders, 39 pts (42%) – non-responders. Number of full responders was higher in pts receiving ruxolitinib as compared to BAT (48.4% vs 12.9%, р<0.0001). QoL in all SF-36 scales was significantly worse in pts with MF comparing to healthy controls; the most pronounced impairment was observed in role physical functioning (39.8 vs 76.9), physical functioning (58.1 vs 85.3) and role emotional functioning (52 vs 77.1); Integral QoL Index in pts was lower than in healthy controls  – 0.3 vs 0.55 (p<0.0001). More than one third of pts had significant or severe QoL impairment: Integral QoL Index was 7 times less as compared to healthy controls. Pts receiving BAT showed more pronounced QoL impairment as compared to pts receiving ruxolitinib: they had worse physical functioning (51.3 vs 74.7), role physical and role emotional functioning (32.2 vs 61.3, 43 vs 75.3), general health (40.9 vs 53.6), vitality (43.9 vs 60.2), social functioning (58.4 vs 81), and mental health (52.1 vs 67), p<0.05; Integral QoL Index was significantly lower – 0.24 vs 0.47 (p<0.0001). Positive changes in health condition measured by PGIC were reported in 67% pts receiving ruxolitinib vs 40% patients receiving BAT (p=0.01). The majority of pts (74%) exhibited at least one moderate-to-severe symptom; the most prevalent among symptoms were fatigue, inactivity, pain in bones/muscles, insomnia, dizziness and feeling of worry. These symptoms were more pronounced in pts on BAT comparing to pts on ruxolitinib (p<0.05). Inactivity appeared to have the most significant negative impact on QoL; multiple linear regression model indicated significant relationship between symptom severity and Integral QoL Index (R-squared=0.65, р<0.001) with the highest regression coefficient for inactivity (Beta-coefficient=0.6).

Conclusion
Patient-reported outcomes such as QoL and symptom burden in MF pts during treatment in a real world setting may be a solid supplement to clinical outcomes and may contribute to better disease control and improved quality of care of this difficult patient population. The data of this real world study demonstrate benefits of ruxolitinib therapy from patient perspective and support the results of clinical studies. 

Session topic: E-poster

Keyword(s): Myelofibrosis, Quality of life
Abstract: PB2025

Type: Publication Only

Background
Myelofibrosis (MF) is associated with poor prognosis, significant symptom burden, and worsened quality of life (QoL). Assessment of patient-reported outcomes is an effective way to identify patients’ risks/benefits of MF treatment. 

Aims
We aimed to study QoL and symptom burden in MF pts in a real world setting as well as to make comparisons in the groups depending on treatment modality.

Methods
A total 93 pts with MF were enrolled in the multicenter real world QoL study – 64 (69%) with primary MF, 14 (15%) post-essential thrombocytopenia MF, 15 (16%) post-polycythemia Vera MF. Median age – 59 years (range 28–90); male/female – 37/56. All pts received the best available treatment (BAT, n=62) or ruxolitinib (n=31) for at least 6 months (median 12 months, range 6–238). High proportion of pts (79%) had IPSS intermediate or high risk. All pts completed the SF-36 QoL questionnaire, symptom assessment questionnaire CSP-MF and Patient Global Impression of Change (PGIC) tool; physicians collected information about disease and treatment from medical records. Integral QoL Index was calculated on the basis of SF-36 QoL questionnaire. For statistical analysis ANOVA, multiple regression and χ2 test were applied.

Results
23 pts (24.7%) from 93 pts were full spleen reduction responders, 31 pts (33.3%) – moderate responders, 39 pts (42%) – non-responders. Number of full responders was higher in pts receiving ruxolitinib as compared to BAT (48.4% vs 12.9%, р<0.0001). QoL in all SF-36 scales was significantly worse in pts with MF comparing to healthy controls; the most pronounced impairment was observed in role physical functioning (39.8 vs 76.9), physical functioning (58.1 vs 85.3) and role emotional functioning (52 vs 77.1); Integral QoL Index in pts was lower than in healthy controls  – 0.3 vs 0.55 (p<0.0001). More than one third of pts had significant or severe QoL impairment: Integral QoL Index was 7 times less as compared to healthy controls. Pts receiving BAT showed more pronounced QoL impairment as compared to pts receiving ruxolitinib: they had worse physical functioning (51.3 vs 74.7), role physical and role emotional functioning (32.2 vs 61.3, 43 vs 75.3), general health (40.9 vs 53.6), vitality (43.9 vs 60.2), social functioning (58.4 vs 81), and mental health (52.1 vs 67), p<0.05; Integral QoL Index was significantly lower – 0.24 vs 0.47 (p<0.0001). Positive changes in health condition measured by PGIC were reported in 67% pts receiving ruxolitinib vs 40% patients receiving BAT (p=0.01). The majority of pts (74%) exhibited at least one moderate-to-severe symptom; the most prevalent among symptoms were fatigue, inactivity, pain in bones/muscles, insomnia, dizziness and feeling of worry. These symptoms were more pronounced in pts on BAT comparing to pts on ruxolitinib (p<0.05). Inactivity appeared to have the most significant negative impact on QoL; multiple linear regression model indicated significant relationship between symptom severity and Integral QoL Index (R-squared=0.65, р<0.001) with the highest regression coefficient for inactivity (Beta-coefficient=0.6).

Conclusion
Patient-reported outcomes such as QoL and symptom burden in MF pts during treatment in a real world setting may be a solid supplement to clinical outcomes and may contribute to better disease control and improved quality of care of this difficult patient population. The data of this real world study demonstrate benefits of ruxolitinib therapy from patient perspective and support the results of clinical studies. 

Session topic: E-poster

Keyword(s): Myelofibrosis, Quality of life

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