Tatiana Ionova

MSc, PhD, DSc, Professor
Chair of Quality of Life Research Unit of St. Petersburg State University Hospital
Chair of Board of the Multinational Center for Quality of Life Research
Saint-Petersburg, Russian Federation
Tatiana Ionova
Tatyana Ionova graduated from St.Petersburg State University. At present she serves as Chief of Quality of Life Monitoring Unit in St.Petersburg State University Hospital, St.Petersburg and Professor of the Department of Hematology and Cellular Therapy, Pirogov National Medical and Surgical Center, Moscow. She is also a Chair of Board of Multinational Center for Quality of Life Research, St.Petersburg.
Since 1995 she initiated and coordinated research in patient-reported outcomes (PRO) in different areas of medicine and contributed a lot to the excellence in quality of life research in Russia and elsewhere. She was a coordinator of national educational programs on quality of life and symptom assessment in health care. She has been actively involved in training patient-reported outcomes research for almost 20 years. She is the Deputy-Editor of the All-Russian scientific journal "Bulletin of Multinational Center for Quality of Life Research". She was invited as a lecturer on PRO assessment for various national and international conferences.
Since 2006 Prof. T. Ionova is Secretary of the EHA Scientific Working Group "Quality of Life and Symptoms" and is actively involved in international projects on PRO in hematology. She is the member of Association of Supportive Care in Oncology (RASSC), European Hematology Association (EHA), International Association of the Study of Lung Cancer (IASLC), International Society for Quality of Life Research (ISOQOL). She advocates for patients with solid tumors, hematological diseases and multiple sclerosis undergoing autologous stem cell transplantation.
She has published more than 350 publications in national and international journals, and is a coauthor of several textbooks on quality of life research. She is the coauthor of the EHA SWG Guidelines "Patient-Reported Outcomes in Hematology" (2012).
Her research interests include implementation of patient-reported outcome (PRO) measures in clinical practice to ensure patient-centered health care, development of PRO instruments, studies on treatment side- effects using PROs, PRO research in rehabilitation and in long-term survivors.

Value of Patient-Reported Outcomes in Multiple Sclerosis Patients
Undergoing Autologous Hematopoietic Stem Cell Transplantation
Sharing the Experience at International Conference in Moscow, Russia
Multiple sclerosis (MS) is a major inflammatory and demyelinating disease of the central nervous system (CNS), which affects mainly young people and leads to progressive quality of life (QoL) deterioration. MS patients suffer from a variety of symptoms which have negative impact on patient's QoL. Most importantly, the level of impact of the wide range of health problems associated with MS needs to be understood in terms of patients' own perceptions of these impacts and the degree to which they affect their lives.

By now there is no known cure for MS. Thus, the goal of treatment is to control symptoms and improve patient's QoL. At present high-dose immunosuppressive therapy with autologous hematopoietic stem cell transplantation (AHSCT) has been used with increasing frequency as a therapeutic option for MS patients. Both disease-free period and improvement of patient's QoL are recognized as important treatment outcomes. With this in mind, evaluation of both clinical and patient-reported outcomes in MS patients after AHSCT is worthwhile.

The information about the disease and its treatment which comes directly from a patient is considered as patient-reported outcomes (PRO). PRO is an umbrella term that is widely used at present. It covers a whole range of potential types of measurement but is used specifically to refer to questionnaires completed by the patient. The most commonly used PRO measures assess QoL and symptoms. QoL is integral characteristics of a physical, psychological, and social functioning of an individual, based on his/her subjective perception" (A. Novik, T. Ionova, P.Kind, 1999). This definition covers 3 major domains of an individual's functioning: physical, psychological and social well-being.

In order to evaluate the efficacy of treatment or rehabilitation of MS patients it is necessary to assess patient's QoL and severity of symptoms. There are several QoL measures which are used

for evaluating QoL in MS. The most wide-spread are generic QoL questionnaires: RAND Short Form-36 (SF-36), EQ-5D and Sickness Impact Profile (SIP). MS-specific measures of QoL include the Functional Assessment of Multiple Sclerosis (FAMS), the Multiple Sclerosis Quality of Life- 54 Instrument (MSQOL-54), and the Disability & Impact Profile (DIP). As for symptom assessment tools it is worth mentioning the Comprehensive Symptom Profile-MS-42 (CSP-MS- 42). This instrument was developed in 2007 by New Jersey Center for Quality of Life and Health Outcome Research (USA) and Multinational Center for QoL Research (Russia). CSP-MS-42 aims to assess the severity of 42 symptoms which are common and most disturbing for MS patients. It consists of numerical analogous scales, scored from "0" (no symptom) to "10" (most expressed symptom).

The model of comprehensive evaluation of outcomes of AHSCT in MS patients, including both clinical and patient-reported outcomes, was developed at the Department of Hematology and Cellular Therapy, Pirogov National Medical and Surgical Center, Moscow. It was implemented in a prospective single center study with the analysis of the safety and efficacy of AHSCT. 502 MS patients (mean age —39 years old; male/female-178/324; meanEDSS=4.0; 257 relapsing/remitting MS, 161 — secondary progressive MS and 84 — primary progressive MS) were included in the analysis. QoL was assessed using RAND SF-36, symptom severity — using CSP-MS-42.

Clinical response in 6 months after AHSCT was observed in almost all patients. At long term follow-up the majority of patients were stable or improved. As for QoL it was significantly compromised before AHSCT. In a year after transplantation definite improvement of QoL parameters was registered with statistically significant changes across all the scales of SF-36, except role emotional functioning (p<0.01). At long-term follow-up (median - 24 months) positive QoL changes preserved: QoL scales were significantly higher than at base-line (p<0.01). Before AHSCT MS patients experienced a wide range of disease-related symptoms. Their severity significantly decreased after transplantation for the majority of items of CSP-MS-42.

Further analysis demonstrated that positive QoL and symptom changes after AHSCT were revealed for different patients' subgroups: for patients with progressive and relapsing disease; for patients with low and high disability according to EDSS; for patients with MS duration less than 5 years and for patients with longer disease duration. This information shows that ASCT is beneficial for different subgroups of MS patients and may be used to the development of recommendations for patient selection for AHSCT.

Thus, AHSCT was accompanied by a significant improvement in patient's QoL and

decrease of symptom burden. Improved QoL was preserved during the entire period of follow-up. AHSCT is beneficial in unfavorable group of MS patients - those with progressive MS, with high disability and with long lasting disease.

Comprehensive evaluation of outcomes of AHSCT is worthwhile. Information about PROs in MS patients undergoing AHSCT may provide valuable information about patient's perspective about the risks/benefits of treatment, patients' needs for rehabilitation and the degree of their recovery.
Tatiana Ionova
St. Petersburg State University Hospital, St Petersburg, Russian Federation