RESUMEN
Differential diagnosis of bone involvement in patients with Gaucher disease can be challenging. Other diseases with similar radiological signs should be ruled out. Here we present a clinical case of tuberculous sacroiliitis in the patient with type I Gaucher disease. Advanced radiological methods of examination are described. Our case report proves the necessity of an individual approach to the management of such cohort of patients. Keywords: Gaucher disease, tuberculosis of bones and joints, differential diagnosis, comprehensive treatment.
Asunto(s)
Enfermedad de Gaucher/diagnóstico por imagen , Radiografía/métodos , Sacroileítis/diagnóstico por imagen , Tuberculosis Osteoarticular/diagnóstico por imagen , Diagnóstico Diferencial , Enfermedad de Gaucher/complicaciones , Humanos , Sacroileítis/complicaciones , Tuberculosis Osteoarticular/complicacionesRESUMEN
Haemostatic management of haemophilia B patients undergoing surgery is critical to patient safety. The aim of this ongoing prospective trial was to investigate the haemostatic efficacy and safety of a recombinant factor IX (rFIX) (Bax326) in previously treated subjects (12-65 years, without history of FIX inhibitors) with severe or moderately severe haemophilia B, undergoing surgical, dental or other invasive procedures. Haemostatic efficacy was assessed according to a predefined scale. Blood loss was compared to the average and maximum blood loss predicted preoperatively. Haemostatic FIX levels were achieved peri- and postoperatively in 100% of subjects (n = 14). Haemostasis was 'excellent' intraoperatively in all patients and postoperatively in those without a drain, and 'excellent' or 'good' at the time of drain removal and day of discharge in those with a drain employed. Following the initial dose, the mean FIX activity level rose from 6.55% to 107.58% for major surgeries and from 3.60% to 81.4% for minor surgeries. Actual vs. predicted blood loss matched predicted intraoperative blood loss but was equal to or higher than (but less than 150%) the maximum predicted postoperative blood loss reflecting the severity of procedure and FIX requirements. There were no related adverse events, severe allergic reactions or thrombotic events. There was no evidence that BAX326 increased the risk of inhibitor or binding antibody development to FIX. BAX326 was safe and effective for peri-operative management of 14 subjects with severe and moderately severe haemophilia B.
Asunto(s)
Pérdida de Sangre Quirúrgica/prevención & control , Coagulantes/uso terapéutico , Factor IX/uso terapéutico , Hemofilia B/tratamiento farmacológico , Hemostasis Quirúrgica/métodos , Procedimientos Quirúrgicos Operativos/métodos , Adolescente , Adulto , Anciano , Inhibidores de Factor de Coagulación Sanguínea/inmunología , Estudios de Casos y Controles , Niño , Coagulantes/efectos adversos , Factor IX/efectos adversos , Femenino , Hemofilia B/inmunología , Hemorragia/prevención & control , Humanos , Masculino , Persona de Mediana Edad , Atención Perioperativa , Cuidados Posoperatorios , Estudios Prospectivos , Proteínas Recombinantes/efectos adversos , Proteínas Recombinantes/uso terapéutico , Adulto JovenRESUMEN
The Pro-FEIBA study reported health-related quality of life (HRQoL) improved following 6-month of Factor Eight Inhibitor Bypassing Activity (FEIBA) prophylaxis. This study investigates whether 12-month of FEIBA prophylaxis improved HRQoL in haemophilia patients with inhibitors. Thirty-six subjects in a 1-year prospective, randomized, open-label, parallel-design study were randomized to prophylaxis (85 ± 15 U kg(-1) every other day) or on-demand treatment. HRQoL was assessed at screening, 6 and 12-month termination using the EQ-5D, Haem-A-QoL, Haemo-QoL and a general pain visual analog scale (VAS). To evaluate changes, paired t-tests and criteria for minimally important differences were applied. Repeated measures regression tested the association between annualized bleeding rate (ABR) and physical HRQoL. At 6 and 12 months, prophylaxis subjects reported clinically meaningful improvement in EQ-5D index (mean improvement, 0.10 and 0.08, respectively) and both clinically meaningful and statistically significant improvements in EQ-VAS scores (16.9 and 15.7, respectively; P < 0.05) vs. baseline. General pain was significantly reduced during prophylaxis at each follow-up (mean improvement, 20.3 and 23.2, respectively; both P <0.05). At 12 months, prophylaxis subjects achieved significant improvements in Haem-A-QoL Total Score and in four domains: Physical Health, Feeling, View, and Work and School (all P < 0.05). No statistically significant changes, except for Haem-A-QoL Physical Health at 6 months, were observed with on-demand treatment. ABR was decreased by 72.5% with prophylaxis vs. on-demand treatment (P = 0.0003) and reduced ABR was associated with better physical HRQoL (P < 0.05). FEIBA prophylaxis significantly reduced ABR and improved HRQoL in inhibitor patients. Subjects with lower ABR reported better physical HRQoL.
Asunto(s)
Factores de Coagulación Sanguínea/uso terapéutico , Coagulantes/administración & dosificación , Factor VIII/administración & dosificación , Estado de Salud , Hemofilia A/tratamiento farmacológico , Calidad de Vida , Adolescente , Adulto , Niño , Humanos , Masculino , Persona de Mediana Edad , Dimensión del Dolor , Estudios Prospectivos , Análisis de Regresión , Encuestas y Cuestionarios , Adulto JovenRESUMEN
The study presents comparative analysis of porous composites made of chitosan, alginate, fibrin with beta-tricalcium phosphate. Histological findings on Wistar rat condyles showed that fibrin-beta-TCP-based composite had the most effective positive biological response.
Asunto(s)
Alginatos/uso terapéutico , Cementos para Huesos/uso terapéutico , Sustitutos de Huesos/uso terapéutico , Fosfatos de Calcio/uso terapéutico , Cementoplastia , Quitosano/uso terapéutico , Fibrina/uso terapéutico , Alginatos/efectos adversos , Alginatos/química , Animales , Cementos para Huesos/efectos adversos , Cementos para Huesos/química , Sustitutos de Huesos/efectos adversos , Sustitutos de Huesos/química , Huesos/efectos de los fármacos , Huesos/lesiones , Fosfatos de Calcio/efectos adversos , Fosfatos de Calcio/química , Quitosano/efectos adversos , Quitosano/química , Fibrina/efectos adversos , Fibrina/química , Ácido Glucurónico/efectos adversos , Ácido Glucurónico/química , Ácido Glucurónico/uso terapéutico , Ácidos Hexurónicos/efectos adversos , Ácidos Hexurónicos/química , Ácidos Hexurónicos/uso terapéutico , Ensayo de Materiales , Porosidad , Ratas , Ratas WistarRESUMEN
BAX326 is a recombinant factor IX (rFIX; nonacog gamma) manufactured without the addition of any materials of human or animal origin, and with two viral inactivation steps (solvent/detergent treatment and 15 nm nanofiltration). The aim of this prospective trial was to investigate the pharmacokinetics, haemostatic efficacy and safety of BAX326 in previously treated patients aged 12-65 years with severe or moderately severe haemophilia B. BAX326 was safe and well tolerated in all 73 treated subjects; adverse events considered related to treatment (2.7% incidence, all non-serious) were transient and mild, and no hypersensitivity reactions, inhibitor formation or thrombotic events were observed. Pharmacokinetic (PK) equivalence (n = 28) between BAX326 and a licensed rFIX was confirmed in terms of the ratio of geometric mean AUC(0-72) h per dose. Twice-weekly prophylaxis [mean duration 6.2 (±0.7) months; 1.8 (±0.1) infusions per week, 49.5 (±4.8) IU kg(-1) per infusion] was effective in preventing bleeding episodes, with a significantly lower (79%, P < 0.001) annualized bleed rate (4.2) compared to an on-demand treatment in a historical control group (20.0); 24 of 56 subjects on prophylaxis (43%) did not bleed throughout the study observation period. Of 249 total acute bleeds, 211 (84.7%) were controlled with one to two infusions of BAX326. Haemostatic efficacy at resolution of bleed was rated excellent or good in 96.0% of all treated bleeding episodes. The results of this study indicate that BAX326 is safe and efficacious in treating bleeds and routine prophylaxis in patients aged 12 years and older with haemophilia B.
Asunto(s)
Factor IX/uso terapéutico , Hemofilia B/tratamiento farmacológico , Proteínas Recombinantes , Adolescente , Adulto , Anciano , Coagulación Sanguínea/efectos de los fármacos , Niño , Factor IX/farmacocinética , Femenino , Hemofilia B/sangre , Humanos , Masculino , Persona de Mediana Edad , Premedicación , Calidad de Vida , Índice de Severidad de la Enfermedad , Resultado del Tratamiento , Adulto JovenRESUMEN
Factor replacement therapy for the treatment of moderate to severe haemophilia A and B can be complicated by the production of inhibitory alloantibodies to factor VIII (FVIII) or factor IX. Treatment with the nanofiltered anti-inhibitor coagulant complex, Factor Eight Inhibitor Bypassing Activity (FEIBA NF), is a key therapeutic option for controlling acute haemorrhages in patients with high-titre inhibitors or low-titre inhibitors refractory to replacement therapy. Given the high risk for morbidity and mortality in haemophilia patients with inhibitors to FVIII or FIX, we conducted this Phase 3 prospective study to evaluate whether prophylaxis with FEIBA NF is a safe and effective treatment option. Over a 1-year period, 17 subjects were treated prophylactically (85 ± 15 U kg(-1) every other day) while 19 subjects were treated on demand. The median (IQR) annualized bleeding rate (ABR) during prophylaxis was 7.9 (8.1), compared to 28.7 (32.3) during on-demand treatment, which amounts to a 72.5% reduction and a statistically significant difference in ABRs between arms (P = 0.0003). Three (17.6%) subjects (ITT) on prophylaxis experienced no bleeding episodes, whereas none treated on demand were bleeding episode-free. Total utilization of FEIBA NF for the treatment of bleeding episodes was significantly higher during on-demand therapy than prophylaxis (P = 0.0067). There were no differences in the rates of related adverse events between arms. This study demonstrates that FEIBA prophylaxis significantly reduces all types of bleeding compared with on-demand treatment, and the safety of prophylaxis is comparable to that of on-demand treatment.
Asunto(s)
Factores de Coagulación Sanguínea/uso terapéutico , Factor IX/uso terapéutico , Factor VIII/uso terapéutico , Hemofilia A/tratamiento farmacológico , Hemofilia B/tratamiento farmacológico , Premedicación , Adolescente , Adulto , Inhibidores de Factor de Coagulación Sanguínea , Factores de Coagulación Sanguínea/administración & dosificación , Factores de Coagulación Sanguínea/efectos adversos , Niño , Factor IX/administración & dosificación , Factor IX/efectos adversos , Factor VIII/administración & dosificación , Factor VIII/efectos adversos , Hemofilia A/sangre , Hemofilia A/complicaciones , Hemofilia B/sangre , Hemofilia B/complicaciones , Hemorragia/etiología , Hemorragia/terapia , Humanos , Masculino , Persona de Mediana Edad , Calidad de Vida , Resultado del Tratamiento , Adulto JovenRESUMEN
Gaucher disease (GD) is an inherited enzymatic defect resulting from a deficiency of acid [3-glucosidase, a lysosomal enzyme involved in the degradation of cell metabolic products. The major clinical manifestations of GD are hepatosplenomegaly, cytopenia, and bony involvement varying from asymptomatic osteopenia to severest osteoporosis and ischemic necrosis to develop irreversible orthopedic defects. Timely enzyme replacement therapy with recombinant glucosidase makes it possible to arrest disease progression and to prevent damage to the vital organs. However, GD in adult patients is frequently diagnosed in the presence of occurring osteoarticular lesions (arthrosis deformans, abnormal fractures). In these instances, besides enzyme replacement therapy, high-quality orthopedic care is required. The description of the case history of a patient undergoing splenectomy in childhood is given as a clinical example of severe osteoarticular lesion in GD and complex differential diagnosis with the intercurrent disease extrapulmonary tuberculosis.
Asunto(s)
Enfermedad de Gaucher/complicaciones , Sacroileítis/complicaciones , Tuberculosis Osteoarticular/complicaciones , Adulto , Antituberculosos/administración & dosificación , Antituberculosos/uso terapéutico , Diagnóstico Diferencial , Terapia de Reemplazo Enzimático , Enfermedad de Gaucher/diagnóstico , Enfermedad de Gaucher/terapia , Glucosilceramidasa/uso terapéutico , Humanos , Masculino , Sacroileítis/diagnóstico , Sacroileítis/terapia , Resultado del Tratamiento , Tuberculosis Osteoarticular/diagnóstico , Tuberculosis Osteoarticular/terapiaRESUMEN
AIM: To evaluate the efficiency of intensive polychemotherapy (PCT) in adult patients having diffuse large B-cell lymphoma (DLBL) of bones and soft tissues with and without poor prognostic factors (PPF). SUBJECTS AND METHODS: Out of 58 enrolled patients, 51 were diagnosed as having DLBL. Burkitt's lymphoma (BL) was diagnosed in 6 patients. One patient had marginal zone B-cell lymphoma. Thirty-five patients with DLBL (10 patients with PPF and 25 without PPF) and 3 patients with BL were treated with the CHOP/R-CHOP regimen. The NHL-BFM-90 program was used in 3 patients with BL and 16 with DLBL (15 patients with PPF and 1 patient without PPF). RESULTS: After radiotherapy, the patient with marginal zone B-cell lymphoma achieved a 20-month remission; three BL patients receiving CHOP died. All the BL patients receiving NHL-BFM-90 achieved complete remissions of 48 to 72 months. In 9 patients having DLBL without PPF, who received CHOP, five-year overall and event-free survival rates were 100%; in 22 patients with PPF, these were 50 and 45%, respectively. In the patients with and without PPF, who received CHOP/R-CHOP, the survival rates differed statistically significantly (p = 0.01; logrank test). In the group of 15 patients having DLBL with PPF, who were treated with the NHL-BFM-90 protocol, 14 achieved an average remission of 17 months. In the patients having DLBL with PPF who used NHF-BFM-90, therapeutic efficiency was significantly higher (p = 0.05; Fisher's exact test). CONCLUSION: Differential therapy for primary lymphomas of bones and soft tissues indicated that the NHL-BFM-90 protocol used in the PPF group was significantly more effective than the CHOP regimens.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Neoplasias Óseas/tratamiento farmacológico , Linfoma de Células B Grandes Difuso/tratamiento farmacológico , Neoplasias de los Tejidos Blandos/tratamiento farmacológico , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Neoplasias Óseas/patología , Supervivencia sin Enfermedad , Femenino , Humanos , Linfoma de Células B Grandes Difuso/patología , Masculino , Persona de Mediana Edad , Pronóstico , Inducción de Remisión/métodos , Neoplasias de los Tejidos Blandos/patología , Tasa de Supervivencia , Factores de Tiempo , Adulto JovenRESUMEN
The study was conducted in 102 patients with hematological disorders admitted to the Research Center of Hematology. It was concluded that more than 50% of them needed oral surgery procedures requiring multidisciplinary approach.
Asunto(s)
Enfermedades Hematológicas/epidemiología , Enfermedades Estomatognáticas/epidemiología , Adulto , Femenino , Enfermedades Hematológicas/terapia , Humanos , Incidencia , Masculino , Persona de Mediana Edad , Federación de Rusia/epidemiología , Adulto JovenRESUMEN
BACKGROUND: Prophylaxis with factor (F)VIII is considered the optimal treatment for managing hemophilia A patients without inhibitors. OBJECTIVES: To compare the efficacy of two prophylaxis regimens (primary outcome) and of on-demand and prophylaxis treatments (secondary outcome), and to continue the evaluation of immunogenicity and overall safety of the ADVATE Antihemophilic Factor (Recombinant), Plasma/Albumin Free Method (rAHF-PFM). PATIENTS/METHODS: Previously on-demand-treated patients aged 7-59 years (n = 66) with FVIII levels ≤ 2% received 6 months of on-demand treatment and then were randomized to 12 months of either standard (20-40 IU kg(-1) every other day) or pharmacokinetic (PK)-tailored (20-80 IU kg(-1) every third day) prophylaxis, both regimens intended to maintain FVIII trough levels at or above 1%. Efficacy was evaluated in terms of annualized bleeding rates (ABRs). As subjects were first treated on-demand and then on prophylaxis, statistical comparisons between these treatments were paired. RESULTS: Twenty-two (33.3%) subjects on prophylaxis experienced no bleeding episodes, whereas none treated on-demand were free from an episode of bleeding. ABRs for the two prophylaxis regimens were comparable, whereas differences between on-demand and either prophylaxis were statistically significant (P < 0.0001): median (interquartile range [IQR]) ABRs were 43.9 (21.9), 1.0 (3.5), 2.0 (6.9) and 1.1 (4.9) during on-demand treatment, standard, PK-tailored and any prophylaxis, respectively. There were no differences in FVIII consumption or adverse event rates between prophylaxis regimens. No subject developed FVIII inhibitors. CONCLUSIONS: The present study demonstrates comparable safety and effectiveness for two prophylaxis regimens and that prophylaxis significantly reduces bleeding compared with on-demand treatment. PK-tailored prophylaxis offers an alternative to standard prophylaxis for the prevention of bleeding.
Asunto(s)
Coagulantes/administración & dosificación , Factor VIII/administración & dosificación , Hemofilia A/tratamiento farmacológico , Hemorragia/prevención & control , Adolescente , Adulto , Niño , Coagulantes/efectos adversos , Coagulantes/farmacocinética , Esquema de Medicación , Monitoreo de Drogas , Europa (Continente) , Factor VIII/efectos adversos , Factor VIII/farmacocinética , Hemofilia A/sangre , Hemofilia A/complicaciones , Hemorragia/sangre , Hemorragia/etiología , Humanos , Masculino , Persona de Mediana Edad , Proteínas Recombinantes/administración & dosificación , Factores de Tiempo , Resultado del Tratamiento , Estados Unidos , Adulto JovenRESUMEN
Case presentation of radical maxillary sinusotomy in chronic sinusitis patient with severe haemophilia A, the paper contains detailed description of the surgical technique, postsurgical care, hemostatic and complementary treatment, as well as anesthesia options making this procedure safe in terms of hemorrhagic complications.
Asunto(s)
Hemofilia A/complicaciones , Sinusitis Maxilar/complicaciones , Sinusitis Maxilar/cirugía , Adulto , Pérdida de Sangre Quirúrgica/prevención & control , Técnicas Hemostáticas , Humanos , Masculino , Hemorragia Posoperatoria/prevención & control , Resultado del TratamientoRESUMEN
Intracranial hemorrhage in patients with chronic autoimmune thrombocytopenic purpura (CATP) is a rare and severe complication of the disease. By taking into account a concomitance of chronic subdural hematoma (CSH) and CATP and no generally accepted approaches to managing the patients with this concomitance, the authors describe a clinical case of mini-invasive CSH drainage in a patient with CATP.
Asunto(s)
Hematoma Subdural Crónico/cirugía , Procedimientos Neuroquirúrgicos , Púrpura Trombocitopénica Idiopática/complicaciones , Esplenectomía , Adulto , Enfermedad Crónica , Glucocorticoides/administración & dosificación , Glucocorticoides/uso terapéutico , Hematoma Subdural Crónico/diagnóstico , Hematoma Subdural Crónico/etiología , Hematoma Subdural Crónico/inmunología , Humanos , Masculino , Recuento de Plaquetas , Púrpura Trombocitopénica Idiopática/diagnóstico , Púrpura Trombocitopénica Idiopática/tratamiento farmacológico , Púrpura Trombocitopénica Idiopática/cirugía , Resultado del TratamientoRESUMEN
AIM: To evaluate efficacy of intensive modified program NHL-BFM-90 (mNHL-BFM-90) in adult poor-prognosis patients with diffuse large B-cell lymphosarcoma (DLBCL) of the bones and soft tissues. MATERIAL AND METHODS: The mNHL-BFM-90 program was used in the treatment of 3 male and 2 female patients aged 17-69 years (median 42 years). Four patients had DLBCL of the bones and one patient--DLBCL of the soft tissues. All the patients had tumors more than 10 cm in size. B-symptoms, a high concentration of lactate dehydrogenase (LDG) were registered in 3 patients. One patient had stage IE by Ann-Arbor, two--stage IIE (involvement of regional lymph nodes), two--stage ME (multiple bone lesions). A total of 4-6 blocks of polychemotherapy according to mNHL-BFM-90 program were performed. RESULTS: Complete remissions were achieved in all the patients. They had no recurrences after 6 to 20 month (median 13 months) follow-up. CONCLUSION: Positive results of the program mNHL-BFM-90 in poor-prognosis patients with DLBCL of the bones and tissues necessitate further studies of this therapy.
Asunto(s)
Protocolos de Quimioterapia Combinada Antineoplásica/uso terapéutico , Neoplasias Óseas/tratamiento farmacológico , Linfoma de Células B Grandes Difuso/tratamiento farmacológico , Neoplasias de los Tejidos Blandos/tratamiento farmacológico , Adolescente , Adulto , Anciano , Protocolos de Quimioterapia Combinada Antineoplásica/administración & dosificación , Neoplasias Óseas/patología , Relación Dosis-Respuesta a Droga , Femenino , Humanos , L-Lactato Deshidrogenasa/sangre , Linfoma de Células B Grandes Difuso/patología , Masculino , Persona de Mediana Edad , Estadificación de Neoplasias , Pronóstico , Inducción de Remisión , Neoplasias de los Tejidos Blandos/patología , Adulto JovenAsunto(s)
Pérdida de Sangre Quirúrgica/prevención & control , Coloides/efectos adversos , Hemofilia A/cirugía , Hemostasis/efectos de los fármacos , Sustitutos del Plasma/efectos adversos , Trombocitopenia/cirugía , Adolescente , Adulto , Factores de Coagulación Sanguínea/análisis , Pruebas de Coagulación Sanguínea , Coloides/uso terapéutico , Dextranos/efectos adversos , Dextranos/uso terapéutico , Gelatina/efectos adversos , Gelatina/uso terapéutico , Hemofilia A/sangre , Humanos , Derivados de Hidroxietil Almidón/efectos adversos , Derivados de Hidroxietil Almidón/uso terapéutico , Sustitutos del Plasma/uso terapéutico , Agregación Plaquetaria/efectos de los fármacos , Estudios Prospectivos , Succinatos/efectos adversos , Succinatos/uso terapéutico , Trombocitopenia/sangre , Resultado del TratamientoRESUMEN
The surgical treatment results of 68 patients with duodenal diverticulum were analyzed, 21 of whom the various interventions on biliary tracts for cholelithic disease and her complications earlier were conducted. Postcholecystectomy syndrome origin was connected with non-diagnosed and non-eliminated duodenal peripapillary diverticulum. The clinical peculiarities, diagnostic algorithm and the complex of preoperative preparation, surgical treatment and complications prophylaxis are adduced. The necessity of associated terminal common bile duct portion correction plus diverticulectomy or intestine exclusion according to A. G. Zemlianoi was suggested.