Prospective Validation of the Italian Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF: Italian) In 186 MPN Patients

Abstract

AbstractAbstract 5060 Background:The Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) is a concise instrument of patient reported outcomes (PRO) designed to assess the unique spectrum of symptoms present in the majority of patients (Mesa et. al. Cancer 2007). We sought to validate the Italian Translation of the MPN-SAF which addresses 19 separate symptoms reported by MPN patients. Methods:Translation: We utilized the standard practice of PRO translation in which 3 independent translations are created by 3 independent translators fluent in both languages. A fourth translator then discussed the translations with the other translators and a consensus translation was obtained.Validation: Patients self completed the MPN-SAF: Italian at the time of a physician office visit and the Italian EORTC-QLQ-C30 (a widely used instrument of PRO for cancer patients) was co-administered for validation purposes. Results:Patients and Symptomatic Burden: 186 patients were prospectively enrolled (ET (N=88; 47%), PV (N=69; 37%) and MF (N=29; 16%)) a median of 6 years (range:0-29) from their diagnosis. Patients were of a median age (62; range 29–91 years) and gender (56% females) typical of the disease. 72% (N=135) had received some form of non-aspirin medical therapy for their disease, and 68% were on therapy at the time of completing the questionnaire. Patients frequently had a history of either thrombotic events (31%) and/or hemorrhagic events (13%). The MPN-SAF measured 19 items in the enrolled patients (data summarized in Table 1).Table 1:Symptomatic burden of MPNs as assessed by the MPN-SAF in 186 Italian patients (results are reported as mean (95% confidence interval of the mean). All Items measured on a 0 (Absent) to 10 (worst imaginable) scale.ET (N=88)PV (N=69)MF (N=29)Total (N=186)Fatigue (BFI Score)2.8 (2.3–3.3)3.0 (2.5–3.5)3.9 (3.0–4.9)3.1 (2.7–3.4)Early Satiety2.2 (1.6–2.9)2.1 (1.4–2.7)2.5 (1.5–3.5)2.2 (1.8–2.6)Abdominal Pain1.5 (1.0–2.0)1.2 (0.8–1.7)2.4 (1.3–3.5)1.5 (1.2–1.8)Abdominal Discomfort2.0 (1.4–2.7)1.9 (1.3–2.5)2.6 (1.4–3.7)2.0 (1.7–2.5)Inactivity1.9 (1.3–2.4)2.1 (1.4–2.7)2.5 (1.3–3.6)2.0 (1.6–2.4)Headache1.2 (0.8–1.7)1.5 (1.0–2.0)1.3 (0.5–2.2)1.4 (1.0–1.7)Concentration Problem2.1 (1.5–2.7)2.4 (1.8–3.1)2.9 (1.7–4.0)2.3 (2.0–2.8)Dizziness2.1 (1.5–2.7)1.7 (1.2–2.2)1.9 (0.9–2.9)1.9 (1.6–2.3)Numbness2.5 (1.8–3.2)2.6 (2.0–3.3)2.2 (1.2–3.2)2.5 (2.1–2.9)Insomnia2.5 (1.8–3.2)2.8 (2.0–3.6)2.2 (1.3–3.0)2.6 (2.1–3.0)Sad Mood2.2 (1.6–2.8)2.5 (1.9–3.2)2.6 (1.5–3.7)2.4 (2.0–2.8)Sexuality Problems3.1 (2.3–3.9)3.3 (2.4–4.2)3.3 (1.9–4.7)3.2 (2.7–3.7)Cough1.3 (0.8–1.9)1.3 (0.8–1.8)1.7 (0.8–2.7)1.4 (1.0–1.7)Night Sweats2.5 (1.8–3.2)2.2 (1.4–2.9)2.6 (1.5–3.6)2.4 (1.9–2.9)Itching1.9 (1.2–2.5)3.7 (2.9–4.5)2.7 (1.5–3.8)2.7 (2.2–3.1)Bone Pain2.4 (1.6–3.1)2.7 (1.9–3.5)2.4 (1.4–3.4)2.5 (2.0–3.0)Fever0.5 (0.2–0.8)0.3 (0.1–0.5)0.6 (0.2–1.1)0.4 (0.2–0.6)Weight Loss1.2 (0.7–1.8)1.0 (0.5–1.4)1.4 (0.4–2.4)1.1 (0.8–1.5)Quality of Life3.3 (2.7–3.9)3.5 (2.9–4.1)4.0 (3.3–4.8)3.5 (3.1–3.9) Validation Analysis:EORTC-QLQ-C30: Consistent with our experience with the MPN-SAF:English, Pearson correlations between MPN-SAF:Italian individual symptom scores and the Italian EORTC-QLQ C30 showed excellent correlations with co-validation questions including fatigue, pain, insomnia, (all p<0.001). Excellent correlations were demonstrated between EORTC-QLQ-C30 subscales and corresponding MPN-SAF measurements. Comparison with MPN-SAF:English:Comparison with 102 patients prospectively completing the MPN-SAF: English (ET=20, PV=23, MF=59) in the USA indicated very strong correlations (when controlling for MPN subtype) in the prevalence of all 19 items assessed and only subtle differences in terms of symptomatic severity for fatigue, itching and insomnia. Conclusions:The MPN-SAF:Italian is an easy to administer, clear, 19-item inventory of patient-reported outcomes that is specific to MPNs. Additionally, the instrument is validated by 1) comparison to previously validated Italian instruments and 2) the correlation with the MPN-SAF:English. Utilization of the instrument in Italian MPN clinical trials will allow for useful comparison to patients completing the MPN-SAF in other countries and will serve as a valuable clinical marker of disease symptom severity. Disclosures:Vannucchi:Novartis: Membership on an entity's Board of Directors or advisory committees. Mesa:SBio: Research Funding; Novartis: Research Funding; Celgene: Research Funding; Incyte: Research Funding; Roche: Research Funding; eisai: Research Funding; telik: Research Funding.