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Congenital Hemophilia A is a complex disease to treat, especially in places without access to hemophilia treatment centers (HTCs). The primary aim of this study was to analyze the outcomes of a cohort of adult people with congenital hemophilia A in an HTC localized in the Bajio region of Mexico. Observational retrospective study of a cohort of 82 adult people with congenital hemophilia A treated in a tertiary-level hospital in the Bajio region of Mexico, between June 2022 and June 2023. The median age of the patients was 29.5 years, 60.9% with severe hemophilia A, 53.6% were under some factor VIII prophylaxis regimen, and 52.4% had home therapy. The median annualized bleeding rate (ABR) was one bleed/year (IQR 0-3 bleeds/year) including a median of zero joint bleeds/year (IQR 0-3 bleeds/year). The presence of high-response inhibitors was detected in 8.5%, with an overall incidence of inhibitors of 14.6% of the cohort. Univariate analysis showed that inhibitors (OR 21.10; CI 95% 1.20-370.3; P = 0.03) and clinical arthropathy (OR 6.14; CI 95% 2.13-17.68; P = 0.001) were significantly higher in severe hemophilia. Clinically significant arthropathy was found in 71.9% of patients. Ultrasonography of the target joints showed that mainly cartilage degeneration was affected. Blood transfusion-associated viral infections were detected in 10.9% of patients. In our HTC, current treatment with hemostatic agents allows adequate control of ABR with acceptable inhibitor rates. However, we still have joint damage in most patients, which is partly explained by the fact that prophylaxis was introduced only in recent years.
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ABSTRACT Introduction: The radiosynovectomy (RS) is one treatment option for recurrent hemarthrosis in patients with hemophilia (PWH). A prospective cohort study was designed to evaluate the effects of the RS on the synovial membrane volume in the ankles and knees of PWH and patient characteristics related to the RS outcome. Methods: In a one-year follow-up, 25 joints of 22 PWH who presented 3 bleeds or more in the same joint over the last 6 months (target joints) were subjected to the RS. Two groups were compared: those who retained target joints following the RS and those who did not (less than 3 bleeds/6 months after the RS). The groups were analyzed according to age, hemophilia type/severity, joint, body mass index (BMI), inhibitor and Hemophilia Joint Health Score 2.1 (HJHS). The magnetic resonance images (MRI) of six ankles and six knees were acquired prior to, and 6 months after, the RS. The synovial membrane volume and arthropathy MRI scale were accessed and volumes were compared and correlated with the Yttrium-90 dose injected. Results: Patients with a mean age of 12 years and a mean HJHS of 6.7 (p < 0.05) retained target joints after the RS. The inhibitor, joint, type/severity of disease and BMI showed no significant differences between groups. The synovial membrane volume had a significant reduction after the RS (p = 0.03), but no correlation with the Yttrium-90 dose. In proportion to the synovial membrane volume, doses injected to the ankles were larger than those injected to the knees. Conclusion: The synovial membrane volume is reduced after the RS, regardless of the effective 90Y dose.
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Humanos , Criança , Adolescente , Adulto , Sinovite , Imageamento por Ressonância Magnética , Sinovectomia , Hemartrose , Hemofilia A , ArtropatiasRESUMO
INTRODUCTION: The radiosynovectomy (RS) is one treatment option for recurrent hemarthrosis in patients with hemophilia (PWH). A prospective cohort study was designed to evaluate the effects of the RS on the synovial membrane volume in the ankles and knees of PWH and patient characteristics related to the RS outcome. METHODS: In a one-year follow-up, 25 joints of 22 PWH who presented 3 bleeds or more in the same joint over the last 6 months (target joints) were subjected to the RS. Two groups were compared: those who retained target joints following the RS and those who did not (less than 3 bleeds/6 months after the RS). The groups were analyzed according to age, hemophilia type/severity, joint, body mass index (BMI), inhibitor and Hemophilia Joint Health Score 2.1 (HJHS). The magnetic resonance images (MRI) of six ankles and six knees were acquired prior to, and 6 months after, the RS. The synovial membrane volume and arthropathy MRI scale were accessed and volumes were compared and correlated with the Yttrium-90 dose injected. RESULTS: Patients with a mean age of 12 years and a mean HJHS of 6.7 (pâ¯<â¯0.05) retained target joints after the RS. The inhibitor, joint, type/severity of disease and BMI showed no significant differences between groups. The synovial membrane volume had a significant reduction after the RS (pâ¯=â¯0.03), but no correlation with the Yttrium-90 dose. In proportion to the synovial membrane volume, doses injected to the ankles were larger than those injected to the knees. CONCLUSION: The synovial membrane volume is reduced after the RS, regardless of the effective 90Y dose.
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INTRODUCTION: Joint affection is a frequent complication of hemophilia. Virtual reality programs offer the patient a dynamic plan with maximum functional benefit. The objective of this work is to evaluate the effect of a rehabilitation program combined with conventional therapy and virtual reality on joint function, in patients with hemophilic arthropathy and the impact on the gait. MATERIAL AND METHODS: Eleven pediatric patients with hemophilia and history of hemophilic arthropathy grade I or II were evaluated. An 8-week rehabilitation program was carried out with Xbox KinectTM and mechanotherapy. Movement analysis laboratory to evaluate the gait pattern and the Joint Health Scale was performed at the beginning and end of the program. RESULTS: A reduction of 50% (p = 0.000), 68% (p = 0.016) and 48% (p = 0.000) were observed in the Total Joint Health score (STA), Global Gait Score (PMG) and Total Score of the Joint Health Scale (P-HJHS-T) respectively. There is a decrease in the support phase and right stride, and increase in cadence, step length, left stride and swing phase. CONCLUSIONS: The overall improvement of the patients makes us conclude that this pilot study opens a window of opportunity to continue the study of an integral rehabilitation program based on the use of virtual reality in pediatric patients with hemophilic arthropathy.
INTRODUCCIÓN: La afección articular es una complicación frecuente de la hemofilia. Los programas de realidad virtual ofrecen al paciente pediátrico un esquema dinámico con máximo beneficio funcional. El objetivo de este trabajo es evaluar el efecto de un programa de rehabilitación combinado con terapia convencional y realidad virtual sobre la función articular en pacientes con artropatía hemofílica y el impacto sobre el patrón de la marcha. MATERIAL Y MÉTODOS: Se evaluaron 11 pacientes pediátricos con hemofilia A o B y antecedente de artropatía hemofílica en grado I o II. Se sometieron a un programa de rehabilitación de ocho semanas con Xbox KinectTM y mecanoterapia. Se utilizó el laboratorio de análisis de movimiento para evaluar el patrón de marcha y la escala de salud articular al inicio y término del programa. RESULTADOS: Se redujo 50% (p = 0.000), 68% (p = 0.016) y 48% (p = 0.000) el puntaje de la escala de salud total articular (STA), puntaje marcha global (PMG) y puntaje total de la escala de salud articular (P-HJHS-T) respectivamente. Se observó disminución en la fase de apoyo y zancada derecha y aumento en cadencia, longitud de paso, zancada izquierda y fase de balanceo. CONCLUSIONES: La mejoría global de los pacientes nos hace concluir que este estudio piloto abre una ventana de oportunidades para continuar el estudio de un programa integral de rehabilitación con base en el uso de la realidad virtual en pacientes pediátricos con artropatía hemofílica.
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Hemofilia A , Realidade Virtual , Criança , Hemartrose , Hemofilia A/complicações , Humanos , Projetos Piloto , Estudos RetrospectivosRESUMO
This study aimed to evaluate synovectomy with samarium-153 hydroxyapatite (153Sm-HA) in synovitis of the elbows and ankles of hemophilic patients. The synovectomy was performed using 185 MBq of 153Sm-HA in 166 joints of 82 hemophilic patients, with a mean age of 24.4 years and follow-up of 12 and 42 months, comprising 63 ankles and 84 elbows. Arthropathy was characterized by recurrent joint bleeding. Episodes of hemarthrosis, use of clotting factors, and pain intensity were evaluated before and after treatment. Scintigraphic controls and adverse effects were also considered. Statistical analyses used P ≤ 0,005 as significant. The results pointed that (a) reduction in hemarthrosis was 78% and 68% in elbows and 82% and 72% in ankles; (b) use of clotting factors was 80% and 70% in elbows and 85% and 75% in ankles; (c) pain was 37% and 34% in elbows and 61% and 57% in ankles, after 12 and 42 months, respectively. Three cases of mild reactive synovitis were observed in ankles and four in elbows. There was no joint effusion in any of the cases. In conclusion, the use of 153Sm-HA in elbows and ankles was effective, very safe, minimally invasive and showed consistency over time, is another material to utilize in median hemophilic joints.
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PURPOSE: Recently, there has been a growing interest in a methodology for dose planning in radiosynoviorthesis to substitute fixed activity. Clinical practice based on fixed activity frequently does not embrace radiopharmaceutical dose optimization in patients. The aim of this paper is to propose and discuss a dose planning methodology considering the radiological findings of interest obtained by three-dimensional magnetic resonance imaging combined with Monte Carlo simulation in radiosynoviorthesis treatment applied to hemophilic arthropathy. METHOD: The parameters analyzed were: surface area of the synovial membrane (synovial size), synovial thickness and joint effusion obtained by 3D MRI of nine knees from nine patients on a SIEMENS AVANTO 1.5 T scanner using a knee coil. The 3D Slicer software performed both the semiautomatic segmentation and quantitation of these radiological findings. A Lucite phantom 3D MRI validated the quantitation methodology. The study used Monte Carlo N-Particle eXtended code version 2.6 for calculating the S-values required to set up the injected activity to deliver a 100 Gy absorbed dose at a determined synovial thickness. The radionuclides assessed were: 90Y, 32P, 188Re, 186Re, 153Sm, and 177Lu, and the present study shows their effective treatment ranges. RESULT: The quantitation methodology was successfully tested, with an error below 5% for different materials. S-values calculated could provide data on the activity to be injected into the joint, considering no extra-articular leakage from joint cavity. Calculation of effective treatment range could assist with the therapeutic decision, with an optimized protocol for dose prescription in RSO. CONCLUSION: Using 3D Slicer software, this study focused on segmentation and quantitation of radiological features such as joint effusion, synovial size, and thickness, all obtained by 3D MRI in patients' knees with hemophilic arthropathy. The combination of synovial size and thickness with the parameters obtained by Monte Carlo simulation such as effective treatment range and S-value, from which is calculated the injected activity, could be used for treatment planning in RSO. Data from this methodology could be a potential aid to clinical decision making by selecting the most suitable radionuclide; justifying the procedure, fractioning the dose, and the calculated injected activity for children and adolescents, considering both the synovial size and thickness.
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Imageamento por Ressonância Magnética , Compostos Radiofarmacêuticos , Planejamento da Radioterapia Assistida por Computador , Humanos , Método de Monte Carlo , Imagens de Fantasmas , Radioisótopos , Dosagem RadioterapêuticaRESUMO
La profilaxis en el tratamiento de la hemofilia ha sido crucial en la mejoría del pronóstico y calidad de vida en las personas con hemofilia (PCH). A pesar de ello, no está globalmente implementado y no ha sido ejecutado satisfactoriamente en Latinoamérica, donde es difícil evaluar la situación, y el manejo de las PCH no se ajusta a los estándares ideales. El grupo GLAITH (Grupo Latino Americano para el Impulso del Tratamiento de la Hemofilia) discutió el problema a través de una encuesta entre sus integrantes. Los hallazgos fueron discutidos en Bogotá en mayo del 2013 en donde los participantes definieron los puntos esenciales a comunicar en un llamado a la acción. Las proporciones de casos de hemofilia A reportados fueron entre 75 y 90% y entre 10 y 25%, los de hemofilia B. La hemofilia grave representó entre el 26 y el 55% de los casos. Un alto porcentaje de PCH tiene artropatía hemofílica. La atención de PCH varía en cada país, sólo se cubre entre el 50 y 60% del tratamiento, que es a demanda en el 85 a 95% de los casos. Sólo 5 a 15% reciben profilaxis, la mayoría secundaria. Pocos países tienen programa nacional o registros homogéneos. En llamado a la acción y conclusión para la región se recomienda: establecimiento de un registro latinoamericano unificado; estudios prospectivos de costo efectividad y evaluación de criterios en profilaxis secundaria; estudios comparativos de calidad de vida, individualización del tratamiento e implementación de la profilaxis en forma global en Latinoamérica.
Prophylactic treatment in the management of hemophilia has been a crucial factor in improving the prognosis and quality of life for people with hemophilia (PCH). However, it is not globally implemented. In Latin America it is difficult to assess the status of PCH and the its management does not conform to ideal standards. The GLAITH group discussed the problem in Latin America. A survey of its members and its findings were discussed at a meeting in Bogota in May 2013. Proportions of hemophilia A and B were 75-90% and 10-25% respectively. Severe hemophilia represents 26-55% of cases. A high percentage of PCH have hemophilic arthropathy. The general care and specific treatments of PCH vary by country, only 50-60% of the treatment is covered and in 85-95% of the cases are performed on an ondemand basis. Just 5-15% receives prophylaxis, most of them secondary. Few countries have a national program or homogeneous records. Finally the GLAITH group proceeded to develop a conclusion and call to action for the region where the following points are recommended: the establishment of a unified Latin American registry; prospective cost-effectiveness studies and evaluation criteria related to secondary prophylaxis; comparative studies of quality of life with and without prophylaxis in the region; promotion of individualization of treatment and, the increase of primary and secondary prophylaxis globally in Latin America.