RESUMEN
The Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS) is a surrogate marker for symptom evaluation in chronic myeloproliferative neoplasms (MPNs). However, insufficient data are available regarding the relationship among the MPN-SAF TSS, JAK2 mutation allele burden, and thrombosis. In this retrospective analysis, we aimed to determine the genetic burdens, clinical features, and relationships with MPN-SAF TSS in MPN patients. One hundred thirty JAK2V617F-positive patients with MPNs were included in our study. We calculated the MPN-SAF TSS for all patients and compared it with their clinical characteristics. Patients with higher JAK2V617F mutation allele burden had higher MPN-SAF TSS values (p=0.008). Patients with thrombosis had higher MPN-SAF TSS than patients without thrombosis (p=0.003). The mean MPN-SAF TSS was higher in patients with primary myelofibrosis compared to those with polycythemia vera and essential thrombocythemia. Thrombosis was associated with increased symptom severity in several domains, including fatigue, abdominal discomfort, inactivity, night sweats, pruritus, weight loss, and early satiety. Additionally, an increase in JAK2 allele burden was observed with higher symptom scores. The MPN-SAF TSS proved to be a reliable tool for assessing symptom burden in Turkish MPN patients. Furthermore, the significant association between thrombosis occurrence and symptom severity suggests that thrombotic events may contribute to symptom development. Notably, increasing JAK2 allele burden was correlated with more severe symptoms, highlighting its potential role in predicting disease burden. This study emphasizes the importance of symptom assessment in MPN patients and supports the incorporation of the MPN-SAF TSS in routine clinical practice to enhance patient care and management.
Asunto(s)
Janus Quinasa 2 , Trastornos Mieloproliferativos , Evaluación de Síntomas , Trombosis , Humanos , Femenino , Masculino , Trastornos Mieloproliferativos/genética , Trastornos Mieloproliferativos/diagnóstico , Trastornos Mieloproliferativos/complicaciones , Persona de Mediana Edad , Janus Quinasa 2/genética , Trombosis/genética , Trombosis/diagnóstico , Trombosis/etiología , Anciano , Estudios Retrospectivos , Adulto , Mutación , Anciano de 80 o más Años , Alelos , Enfermedad CrónicaRESUMEN
BACKGROUND: Approximately 50% of patients with polycythemia vera (PV) have PV-related symptoms at diagnosis; these symptoms might develop or worsen with time. Symptoms have been shown to negatively affect quality of life and interfere with daily activities. To our knowledge, an analysis to evaluate the relationship between blood count control and symptoms has not been published. PATIENTS AND METHODS: The Prospective Observational Study of Patients with Polycythemia Vera in US Clinical Practices (REVEAL; NCT02252159) is a multicenter, noninterventional, nonrandomized prospective observational study of patients with PV in the United States. Patients included were required to have a complete blood count result within 30 days before completing the at-enrollment Myeloproliferative Neoplasm Self-Assessment Form Total Symptom Score (MPN-SAF TSS). Symptom severity was compared between those who had blood count control versus those who did not. RESULTS: At the time of enrollment, 1714 patients (94.5%) were being managed with cytoreductive therapy; 468 patients (25.8%) had complete hematologic remission (CHR), 1614 patients (89.0%) had ≥1 controlled blood count, and 1122 patients (61.9%) had ≥2 controlled blood counts. Mean MPN-SAF TSSs were similar across patients in different blood count control groups. Fatigue was the most frequently reported symptom. The severity of individual symptoms, except those of pruritus and night sweats, was not affected by CHR or the number of blood counts that were controlled. CONCLUSION: Symptom burden in patients with PV can persist despite control of blood counts, which suggests some discordance between laboratory values and symptom burden. Consequently, regular monitoring of symptom burden should be factored into the assessment of disease control.
Asunto(s)
Recuento de Células Sanguíneas , Policitemia Vera/sangre , Policitemia Vera/diagnóstico , Adulto , Anciano , Anciano de 80 o más Años , Antineoplásicos/administración & dosificación , Antineoplásicos/efectos adversos , Antineoplásicos/uso terapéutico , Manejo de la Enfermedad , Femenino , Humanos , Masculino , Persona de Mediana Edad , Policitemia Vera/complicaciones , Policitemia Vera/terapia , Estudios Prospectivos , Índice de Severidad de la Enfermedad , Evaluación de Síntomas , Resultado del Tratamiento , Estados Unidos , Adulto JovenRESUMEN
OBJECTIVE: To investigate the intensity of burdensome symptoms using self-assessment MPN-SAF TSS in patientswith radiation-associated and spontaneous myeloproiliferative neoplasms (MPNs). MATERIALS AND METHODS: The study included 89 patients with radiation-associated and spontaneous MPNs, the bur-densome symptoms of MPN were determined using MPN-SAF TSS. RESULTS: The average score for complaints in patients with radiation-associated MPNs was significantly higher thanin patients with spontaneous MPNs - 43.46 and 25.04 points, respectively (p = 0.003). MPN patients classified bysubtypes also showed differences regarding intensity of burdensome MPN symptoms, demonstrating significantlyhigher average score of complaints among primary myelofibrosis patients (35.60), compared to polycythemia vera(29.60) and essential thrombocythemia (18.05) patients, (p = 0.005). Our study did not reveal any influence of theJAK2 V617F mutation on MPN burdensome symptoms intensity in MPN patients. CONCLUSIONS: We demonstrated a higher intensity of the MPN burdensome symptoms determined by the optimizedself-assessment MPN-SAF TSS in patients with radiation-associated, and in primary myelofibrosis patients, indicat-ing increased severity of patient's general conditions at the stage of diagnosis verification. It is advisable to usethe optimized MPN-SAF TSS at the moment of molecular genetic testing during the diagnosis of MPN for selectionor modifying treatment strategies in order to achieve better quality of life for patients.
Asunto(s)
Accidente Nuclear de Chernóbil , Exposición a Riesgos Ambientales/efectos adversos , Policitemia Vera/fisiopatología , Mielofibrosis Primaria/fisiopatología , Exposición a la Radiación/efectos adversos , Trombocitemia Esencial/fisiopatología , Adulto , Anciano , Femenino , Expresión Génica , Humanos , Janus Quinasa 2/genética , Masculino , Persona de Mediana Edad , Mutación , Policitemia Vera/etiología , Policitemia Vera/genética , Policitemia Vera/psicología , Mielofibrosis Primaria/etiología , Mielofibrosis Primaria/genética , Mielofibrosis Primaria/psicología , Calidad de Vida/psicología , Dosis de Radiación , Radiación Ionizante , Autoevaluación (Psicología) , Índice de Severidad de la Enfermedad , Trombocitemia Esencial/etiología , Trombocitemia Esencial/genética , Trombocitemia Esencial/psicología , UcraniaRESUMEN
Resumen Objetivo: Validar en Colombia la escala abreviada de síntomas representativos de neoplasias mieloproliferativas para su posterior uso en investigación y aplicación en el ámbito clínico. Materiales y métodos: Se realizó la adaptación transcultural de la escala MPN-SAF-TSS (por sus siglas en inglés: Mieloproliferative Neoplasm Symptom Assesment Form-Total Sympton Score) a español y al contexto colombiano, con análisis estadísticos de validez y confiabilidad con un tamaño de muestra determinado para cada propiedad psicométrica. Resultados: Con base en las propiedades psicométricas se logró verificar la confiabilidad y la estructura interna de la escala con alfa de Cronbach de 0,8552, lo que denota una buena consistencia interna; se obtuvo un coeficiente de correlación de concordancia de Lin de 0,765 que muestra una buena confiabilidad. El análisis factorial mostró múltiples valores de coeficiente de correlación por encima de 0,3, lo que indica que las variables de la escala miden factores comunes; la validez de criterio concurrente determina que la escala mide lo que debe medir con un adecuado coeficiente de correlación de Spearman de -0,71 y -0,73 al confrontarla con las escalas FACIT-Fatigue y Fact-Lym respectivamente. Conclusión: La escala MPN-SAF-TSS adaptada a español y al contexto colombiano es un instrumento válido para la evaluación de síntomas en pacientes con neoplasias mieloproliferativas y puede ser utilizado en la práctica clínica o en estudios clínicos por sus adecuadas propiedades psicométricas.
Abstract Objective: To validate in Colombia the abbreviated scale of representative symptoms of myeloproliferative neoplasms, for further use in research and for application in the clinical setting. Materials and methods: A cross-cultural adaptation of the MPN-SAF TSS-scale (mieloproliferative neoplasm symptom assessment form total symptom score) to Colombian Spanish using statistical validity and reliability analysis with a specific sample size for each psychometric property. Results. Based on the psychometric properties, the verification of reliability and internal structure with a Cronbach's alpha scale of 0.8552 was achieved, denoting good internal consistency. A Lin's concordance correlation coefficient of 0.765 was obtained, showing good reliability; factor analysis showed multiple correlation coefficient values above 0.3, indicating that the scale variables measure common factors. Concurrent validity determines that the scale measures what it should measure with an adequate Spearman correlation coefficient of -0.71 and -0.73 when confronted with the FACIT-Fatigue and Fact-Lym scales, respectively. Conclusion: The MPN-SAF-TSS scale adapted to Colombian Spanish is a valid assessment instrument for symptoms in patients with myeloproliferative neoplasms and can be used in clinical practice or trials due to its adequate psychometric properties.
Resumo Objetivo: Validar na Colômbia, a escala abreviada de sintomas representativos de neoplasias mielo-proliferativas, para u seu posterior uso em investigação e a sua aplicação no âmbito clínico. Materiais e métodos: Realizou-se a adaptação transcultural da escala MPN-SAF-TSS (pelas suas siglas em inglês mieloproliferative neoplasm symptom assesment form total sympton score) ao espanhol colombiano, com análises estadísticas de validez e confiabilidade com um tamanho de amostra determinado para cada propriedade psicométrica. Resultados: Com base nas propriedades psicomé-tricas conseguiu-se verificar a confiabilidade e a estrutura interna da escala com alfa de Cronbach de 0,8552, o que denota uma boa consistência interna; se obteve um coeficiente de correlação de concordância de Lin de 0,765 mostrando uma boa confiabilidade; a análise fatorial mostrou múltiplos valores de coeficiente de correlação por acima de 0.3 indicando que as variáveis da escala medem fatores comuns, a validez de critério concorrente determina que a escala mede o que deve medir, com um adequado coeficiente de correlação de Spearman de -0,71 e -0,73 ao confrontá-la com as escalas FACIT-Fatigue e Fact-Lym respectivamente. Conclusão: A escala MPN-SAF TSS adaptada ao espanhol colombiano, é um instrumento válido para a avaliação de sintomas em pacientes com neoplasias mieloproliferativas e pode ser utilizado na prática clínica ou em estudos clínicos pelas suas adequadas propriedades psicométricas.