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1.
Skeletal Radiol ; 2024 Jan 04.
Artículo en Inglés | MEDLINE | ID: mdl-38172249

RESUMEN

The future will bring increasing demands for specialty training of MSK radiologists. These will include requirements for further standardization of curriculum, educational methods, and documentation. This will place further burdens (both temporal and financial) on the fellowship programs and may lead to termination of some programs. There is a risk that increasingly bureaucratic training requirements may "crowd out" the basic conceptual education necessary for the continued growth and relevance of the subspecialty. Educators must find ways to incorporate these aspects into programs even when they are not mandated.

2.
Skeletal Radiol ; 51(11): 2217-2221, 2022 Nov.
Artículo en Inglés | MEDLINE | ID: mdl-35301556

RESUMEN

The use of computer-navigated, robotic-assisted total knee arthroplasty (TKA) is expanding as the result of widespread efforts to improve the precision of implant placement and to restore mechanical, anatomic, and kinematic alignment. The procedure requires placement of femoral and tibial pins to mount the robotic tracking system. Fractures through tracking pin sites following robotic-assisted TKA are rare; only 30 cases have been reported to our knowledge. It is probable that this complication will become more frequent as the use of robotic-assisted TKAs continues to grow. We report the case of a 67-year-old female who suffered a fracture of the femur through two pin sites 3 months after a robotic-assisted TKA. We believe that this report is the first case in the radiologic literature. Our case demonstrates some of the difficulties of recognizing this complication and raises questions about appropriate post-operative imaging.


Asunto(s)
Artroplastia de Reemplazo de Rodilla , Fracturas del Fémur , Procedimientos Quirúrgicos Robotizados , Cirugía Asistida por Computador , Anciano , Artroplastia de Reemplazo de Rodilla/métodos , Clavos Ortopédicos/efectos adversos , Femenino , Fracturas del Fémur/diagnóstico por imagen , Fracturas del Fémur/cirugía , Fémur/cirugía , Humanos , Articulación de la Rodilla/cirugía , Procedimientos Quirúrgicos Robotizados/efectos adversos , Procedimientos Quirúrgicos Robotizados/métodos , Cirugía Asistida por Computador/métodos
3.
AJR Am J Roentgenol ; 217(5): 1057-1068, 2021 11.
Artículo en Inglés | MEDLINE | ID: mdl-33336581

RESUMEN

Vertebral discitis-osteomyelitis is an infection of the intervertebral disk and vertebral bodies that may extend to adjacent paraspinal and epidural soft tissues. Its incidence is increasing, likely because of improved treatments and increased life expectancy for patients with predisposing chronic disease and increased rates of IV drug use and intravascular intervention. Because blood cultures are frequently negative in patients with vertebral discitis-osteomyelitis, biopsy is often indicated to identify a causative microorganism for targeted antimicrobial therapy. The reported yield of CT-guided percutaneous sampling is 31-91%, which is lower than the reported yield of open biopsy of 76-91%. However, the less invasive approach may be favored given its relative safety and low cost. If paravertebral fluid collections are present, CT-guided aspiration should be performed. If aspiration is unsuccessful or no paravertebral fluid collections are present, CT-guided percutaneous biopsy should be performed, considering technical factors (e.g., anatomic approach, needle selection, and needle angulation) that may improve microbiologic yield. Although antimicrobial therapy should be withheld for 1-2 weeks before biopsy if clinically feasible, biopsy may still be performed without stopping antimicrobial therapy if needed. Because of the importance of targeted antimicrobial therapy, repeat biopsy should be considered after 72 hours if initial biopsy does not identify a pathogen.


Asunto(s)
Discitis/diagnóstico por imagen , Biopsia Guiada por Imagen/métodos , Vértebras Lumbares/diagnóstico por imagen , Osteomielitis/diagnóstico por imagen , Anciano , Antibacterianos/uso terapéutico , Discitis/tratamiento farmacológico , Discitis/microbiología , Discitis/patología , Humanos , Biopsia Guiada por Imagen/efectos adversos , Vértebras Lumbares/microbiología , Vértebras Lumbares/patología , Masculino , Osteomielitis/tratamiento farmacológico , Osteomielitis/microbiología , Osteomielitis/patología , Tomografía Computarizada por Rayos X
4.
Skeletal Radiol ; 48(10): 1623-1628, 2019 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-30850870

RESUMEN

Osteoblastoma is a rare, benign primary tumor of bone, accounting for < 1% of all bone tumors. We report the case of a 27-year-old female who developed pain and swelling five and a half years after a clavicular fracture and was subsequently found to have an osteoblastoma arising at the fracture site. This is the first reported case of an osteoblastoma developing after a fracture, although osteoid osteomas, which are histologically indistinguishable from osteoblastomas, have been reported at prior fracture sites. This report demonstrates that secondary neoplasms such as osteoblastomas should be considered in the differential diagnosis for pain at a healed fracture site recurring years after the initial trauma.


Asunto(s)
Neoplasias Óseas/diagnóstico por imagen , Clavícula/diagnóstico por imagen , Clavícula/lesiones , Fracturas Óseas , Osteoblastoma/diagnóstico por imagen , Adulto , Biopsia , Neoplasias Óseas/patología , Neoplasias Óseas/cirugía , Clavícula/cirugía , Femenino , Humanos , Imagen por Resonancia Magnética , Osteoblastoma/patología , Osteoblastoma/cirugía , Tomografía Computarizada por Rayos X
5.
Radiology ; 284(3): 766-776, 2017 09.
Artículo en Inglés | MEDLINE | ID: mdl-28430557

RESUMEN

Purpose To quantify the effect of a comprehensive, long-term, provider-led utilization management (UM) program on high-cost imaging (computed tomography, magnetic resonance imaging, nuclear imaging, and positron emission tomography) performed on an outpatient basis. Materials and Methods This retrospective, 7-year cohort study included all patients regularly seen by primary care physicians (PCPs) at an urban academic medical center. The main outcome was the number of outpatient high-cost imaging examinations per patient per year ordered by the patient's PCP or by any specialist. The authors determined the probability of a patient undergoing any high-cost imaging procedure during a study year and the number of examinations per patient per year (intensity) in patients who underwent high-cost imaging. Risk-adjusted hierarchical models were used to directly quantify the physician component of variation in probability and intensity of high-cost imaging use, and clinicians were provided with regular comparative feedback on the basis of the results. Observed trends in high-cost imaging use and provider variation were compared with the same measures for outpatient laboratory studies because laboratory use was not subject to UM during this period. Finally, per-member per-year high-cost imaging use data were compared with statewide high-cost imaging use data from a major private payer on the basis of the same claim set. Results The patient cohort steadily increased in size from 88 959 in 2007 to 109 823 in 2013. Overall high-cost imaging utilization went from 0.43 examinations per year in 2007 to 0.34 examinations per year in 2013, a decrease of 21.33% (P < .0001). At the same time, similarly adjusted routine laboratory study utilization decreased by less than half that rate (9.4%, P < .0001). On the basis of unadjusted data, outpatient high-cost imaging utilization in this cohort decreased 28%, compared with a 20% decrease in statewide utilization (P = .0023). Conclusion Analysis of high-cost imaging utilization in a stable cohort of patients cared for by PCPs during a 7-year period showed that comprehensive UM can produce a significant and sustained reduction in risk-adjusted per-patient year outpatient high-cost imaging volume. © RSNA, 2017.


Asunto(s)
Diagnóstico por Imagen , Pacientes Ambulatorios/estadística & datos numéricos , Atención Primaria de Salud , Diagnóstico por Imagen/economía , Diagnóstico por Imagen/estadística & datos numéricos , Femenino , Humanos , Masculino , Persona de Mediana Edad , Médicos de Atención Primaria/estadística & datos numéricos , Atención Primaria de Salud/economía , Atención Primaria de Salud/estadística & datos numéricos , Estudios Retrospectivos
7.
Skeletal Radiol ; 46(3): 295-298, 2017 Mar.
Artículo en Inglés | MEDLINE | ID: mdl-28012122

RESUMEN

A look back at Skeletal Radiology in 2016 reveals a sizable number of publications that significantly advanced the state of knowledge about diseases of the musculoskeletal system. This review summarizes the content of some of the most intriguing papers of the year.


Asunto(s)
Sistema Musculoesquelético/diagnóstico por imagen , Publicaciones Periódicas como Asunto/estadística & datos numéricos , Edición/estadística & datos numéricos , Radiología , Humanos
8.
Skeletal Radiol ; 46(10): 1367-1378, 2017 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-28660403

RESUMEN

OBJECTIVE: Hemispherical spondylosclerosis (HS) is a rare degenerative entity characterized by dome-shaped sclerosis of a single vertebral body that may pose a diagnostic dilemma. The goal of this study was to describe the MR imaging features of HS. MATERIALS AND METHODS: We identified spine radiographs and CT examinations of subjects with HS who also had MR imaging for correlation. Two musculoskeletal radiologists independently assessed sclerosis characteristics, presence of endplate erosions, marrow signal intensity, and disk degeneration (Pfirrmann scale). RESULTS: We identified 11 subjects (six males, five females, mean 48 ± 10 years) with radiographic/CT findings of HS. The most commonly affected vertebral body was L4 (6/11; 55%). On MR imaging, variable signal intensity was noted, being most commonly low on T1 (8/11, 73%) and high on fat-suppressed T2-weighted (8/11, 73%) images. In two subjects, diffuse post-contrast enhancement was seen in the lesion. Moderate disk degeneration and endplate bone erosions adjacent to sclerosis were present in all subjects. Erosions of the opposite endplate were present in two subjects (2/11, 18%). CT data from nine subjects showed the mean attenuation value of HS was 472 ± 96 HU. CONCLUSIONS: HS appearance on MR imaging is variable and may not correlate with the degree of sclerosis seen on radiographs or CT. Disk degenerative changes and asymmetric endplate erosions are consistent markers of HS.


Asunto(s)
Imagen por Resonancia Magnética/métodos , Espondilosis/diagnóstico por imagen , Espondilosis/patología , Medios de Contraste , Diagnóstico Diferencial , Femenino , Humanos , Masculino , Persona de Mediana Edad , Tomografía Computarizada por Rayos X
9.
Histopathology ; 69(4): 614-23, 2016 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-27028835

RESUMEN

AIMS: To assess the clinical and histopathological spectrum of primary intra-articular sarcomas. METHODS AND RESULTS: Fifteen patients were identified as having primary intra-articular sarcomas and the clinical and pathological features were evaluated. There were nine males and six females who ranged in age from 16 to 84 (mean 44) years. All tumours originated in the knee joint. The pathological diagnoses included: five synovial sarcoma, three extraskeletal myxoid chondrosarcomas, two high-grade myxofibrosarcoma (one conventional, one epithelioid), two undifferentiated pleomorphic sarcoma (one with giant cells) and one each myxoinflammatory fibroblastic sarcoma, conventional hyaline chondrosarcoma, and high-grade myofibroblastic sarcoma. All tumours were treated by segmental resection or amputation. Adjuvant therapy was given in selected cases. Follow-up ranged from 11 to 150 months. Of patients with follow-up, two died of disease; one developed pulmonary metastases after 6 years and was then lost to follow-up. Nine patients were alive and free of disease 12-150 months after diagnosis. CONCLUSIONS: Primary sarcomas of the joints are very rare and most frequently affect the knee. Our experience indicates that synovial sarcoma and extraskeletal myxoid chondrosarcoma are the most common intra-articular sarcomas.


Asunto(s)
Artropatías/patología , Articulación de la Rodilla/patología , Sarcoma/patología , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Femenino , Humanos , Masculino , Persona de Mediana Edad , Adulto Joven
10.
Radiographics ; 36(6): 1871-1887, 2016 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-27726750

RESUMEN

Metabolic bone diseases are a diverse group of diseases that result in abnormalities of (a) bone mass, (b) structure mineral homeostasis, (c) bone turnover, or (d) growth. Osteoporosis, the most common metabolic bone disease, results in generalized loss of bone mass and deterioration in the bone microarchitecture. Impaired chondrocyte development and failure to mineralize growth plate cartilage in rickets lead to widened growth plates and frayed metaphyses at sites of greatest growth. Osteomalacia is the result of impaired mineralization of newly formed osteoid, which leads to characteristic Looser zones. Hypophosphatasia is a congenital condition of impaired bone mineralization with wide phenotypic variability. Findings of hyperparathyroidism are the result of bone resorption, most often manifesting as subperiosteal resorption in the hand. Renal osteodystrophy is the collection of skeletal findings observed in patients with chronic renal failure and associated secondary hyperparathyroidism and can include osteopenia, osteosclerosis, and "rugger jersey spine." Hypoparathyroidism is most commonly due to iatrogenic injury, and radiographic findings of hypoparathyroidism reflect an overall increase in bone mass. Thyroid hormone regulates endochondral bone formation; and congenital hypothyroidism, when untreated, leads to delayed bone age and absent, irregular, or fragmented distal femoral and proximal tibial epiphyses. Soft-tissue proliferation of thyroid acropachy is most often observed in the hands and feet. The findings of acromegaly are due to excess growth hormone secretion and therefore proliferation of the bones and soft tissues. Vitamin C deficiency, or scurvy, impairs posttranslational collagen modification, leading to subperiosteal hemorrhage and fractures. ©RSNA, 2016.


Asunto(s)
Enfermedades Óseas Metabólicas/diagnóstico por imagen , Huesos/diagnóstico por imagen , Errores Diagnósticos/prevención & control , Tomografía Computarizada por Rayos X/métodos , Diagnóstico Diferencial , Medicina Basada en la Evidencia , Humanos , Aumento de la Imagen/métodos , Posicionamiento del Paciente/métodos
11.
BMC Med Imaging ; 16(1): 53, 2016 09 08.
Artículo en Inglés | MEDLINE | ID: mdl-27609115

RESUMEN

BACKGROUND: Chordomas are rare malignancies that primarily affect adults, but also rarely affect pediatric patients. We compared the imaging appearance, demographic and anatomic distributions of adult and pediatric chordomas in a large cohort. METHODS: We performed a retrospective review of medical records of 220 subjects with histologically confirmed chordomas of the axial skeleton and pre-treatment magnetic resonance imaging studies. Age, sex, type of chordoma (conventional, chondroid or dedifferentiated), the anatomic location of the chordoma, as well as whether the lesion was primarily extra-osseous were recorded. Pediatric subjects were less than 21 years at the time of diagnosis. Binomial two-sample tests of proportions and Fisher's exact tests were used to compare proportions between the pediatric and adult subjects. RESULTS: Fifty six pediatric subjects (58.9 % female) and 164 adult subjects (42.1 % female) were identified. The proportion of female subjects with chordomas was significantly higher in the pediatric cohort compared to the adult cohort (P = 0.04). Most chordomas occur in Caucasians, however African-Americans were more represented in the pediatric cohort than in the adult cohort (P = 0.01). 69.6 % (39/56) of the pediatric chordomas involved the clivus/skull base and cervical spine compared to 29.3 % (48/164) of the adult chordomas (P = 1.99 × 10(-7)). Only 1.8 % (1/56) of the pediatric chordomas was in the sacrococcygeal region compared to 36.0 % (59/164) of the adult chordomas (P = 2.55 × 10(-8)). In cases where pre-treatment imaging was available, 93.8 % (16/17) of pediatric chordomas were predominantly extra-osseous compared to 76.7 % (46/60) of adult chordomas (P = 0.17). CONCLUSIONS: Pediatric chordomas more often affect females and occur most frequently at the craniocervical junction with decrease in incidence distally in the spine, whereas adult chordomas most frequently involve the craniocervical and sacrococcygeal regions.


Asunto(s)
Negro o Afroamericano/estadística & datos numéricos , Cordoma/diagnóstico por imagen , Imagen por Resonancia Magnética/métodos , Población Blanca/estadística & datos numéricos , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Niño , Preescolar , Cordoma/etnología , Femenino , Humanos , Lactante , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Distribución por Sexo , Estados Unidos/etnología , Adulto Joven
12.
Skeletal Radiol ; 45(12): 1687-1693, 2016 Dec.
Artículo en Inglés | MEDLINE | ID: mdl-27726015

RESUMEN

OBJECTIVE: The objective of this study is to determine how a limited protocol MR examination compares to a full conventional MR examination for the detection of non-degenerative pathology such as acute fracture, infection, and malignancy. MATERIALS AND METHODS: A sample of 349 non-contrast MR exams was selected retrospectively containing a 3:1:1:1 distribution of negative/degenerative change only, acute fracture, infection, and malignancy. This resulted in an even distribution of pathology and non-pathology. A limited protocol MR exam was simulated by extracting T1-weighted sagittal and T2-weighted fat-saturated (or STIR) sagittal sequences from each exam and submitting them for blinded review by two experienced musculoskeletal radiologists. The exams were evaluated for the presence or absence of non-degenerative pathology. Interpretation of the limited exam was compared to the original report of the full examination. If either reader disagreed with the original report, the case was submitted for an unblinded adjudication process with the participation of a third musculoskeletal radiologist to establish a consensus diagnosis. RESULTS: There were five false negatives for a sensitivity of 96.9 % for the limited protocol MR exam. Infection in the psoas, paraspinal muscles, and sacroiliac joint, as well as acute fractures in transverse processes and sacrum were missed by one or more readers. No cases of malignancy were missed. Overall diagnostic accuracy was 96.0 % (335/349). CONCLUSIONS: MR imaging of the lumbar spine limited to sagittal T1-weighted and sagittal T2 fat-saturated (or STIR) sequences has high sensitivity for the detection of acute fracture, infection, or malignancy compared to a conventional MR examination.


Asunto(s)
Infecciones/diagnóstico por imagen , Vértebras Lumbares/diagnóstico por imagen , Imagen por Resonancia Magnética , Neoplasias/diagnóstico por imagen , Fracturas de la Columna Vertebral/diagnóstico por imagen , Adulto , Anciano , Anciano de 80 o más Años , Femenino , Humanos , Región Lumbosacra , Masculino , Persona de Mediana Edad , Estudios Retrospectivos , Sacro , Sensibilidad y Especificidad , Adulto Joven
13.
Skeletal Radiol ; 44(6): 875-81, 2015 Jun.
Artículo en Inglés | MEDLINE | ID: mdl-25503857

RESUMEN

Osteoid osteoma is a relatively common, benign, painful tumor of bone. It is widely believed to run a course culminating in spontaneous regression. The tumor can usually be eliminated by excision or ablation, although it may recur locally. Although management has classically been surgical, thermocoagulation via percutaneously delivered radiofrequency energy has demonstrated excellent results, typically resulting in durable response following a single treatment. Here, we present an unusual case of serially recurrent pathologically proven pediatric osteoid osteoma, treated by radiofrequency ablation five times over the course of 11 years. Limitations of RF ablation of osteoid osteoma and possible factors predisposing to incomplete treatment or recurrence are discussed.


Asunto(s)
Neoplasias Óseas/diagnóstico por imagen , Neoplasias Óseas/cirugía , Recurrencia Local de Neoplasia/diagnóstico por imagen , Recurrencia Local de Neoplasia/prevención & control , Osteoma Osteoide/diagnóstico por imagen , Osteoma Osteoide/cirugía , Tibia , Niño , Preescolar , Humanos , Lactante , Estudios Longitudinales , Masculino , Tomografía Computarizada por Rayos X , Resultado del Tratamiento
14.
Skeletal Radiol ; 44(12): 1795-803, 2015 Dec.
Artículo en Inglés | MEDLINE | ID: mdl-26338069

RESUMEN

OBJECTIVE: To study non-diagnostic CT-guided musculoskeletal biopsies and take steps to minimize them. Specifically we asked: (1) What malignant diagnoses have a higher non-diagnostic rate? (2) What factors of a non-diagnostic biopsy may warrant more aggressive pursuit? (3) Do intra-procedural frozen pathology (FP) or point-of-care (POC) cytology reduce the non-diagnostic biopsy rate? MATERIALS AND METHODS: This study was IRB-approved and HIPAA-compliant. We retrospectively reviewed 963 consecutive CT-guided musculoskeletal biopsies. We categorized pathology results as malignant, benign, or non-diagnostic and recorded use of FP or POC cytology. Initial biopsy indication, final diagnosis, method of obtaining the final diagnosis of non-diagnostic biopsies, age of the patient, and years of biopsy attending experience were recorded. Groups were compared using Pearson's χ(2) test or Fisher's exact test. RESULTS: In all, 140 of 963 (15%) biopsies were non-diagnostic. Lymphoma resulted in more non-diagnostic biopsies (P < 0.0001). While 67% of non-diagnostic biopsies yielded benign diagnoses, 33% yielded malignant diagnoses. Patients whose percutaneous biopsy was indicated due to the clinical context without malignancy history almost always generated benign results (96%). Whereas 56% of biopsies whose indication was an imaging finding of a treatable lesion were malignant, 20% of biopsies whose indication was a history of malignancy were malignant. There was no statistically significant difference in the nondiagnostic biopsy rates of pediatric versus adult patients (P = 0.8) and of biopsy attendings with fewer versus more years of experience (P = 0.5). The non-diagnostic rates of biopsies with FP (8%), POC cytology (25%), or neither (24%) were significantly different (P < 0.0001). CONCLUSION: Lymphoma is the malignant diagnosis most likely to result in a non-diagnostic biopsy. If the clinical and radiologic suspicion for malignancy is high, repeat biopsy is warranted. If the clinical context suggests a benign lesion, a non-diagnostic biopsy may be considered reassuring. Frozen pathology may decrease the non-diagnostic biopsy rate.


Asunto(s)
Neoplasias Óseas/epidemiología , Neoplasias Óseas/patología , Biopsia Guiada por Imagen/estadística & datos numéricos , Neoplasias de los Tejidos Blandos/epidemiología , Neoplasias de los Tejidos Blandos/patología , Tomografía Computarizada por Rayos X/estadística & datos numéricos , Adolescente , Adulto , Anciano , Biopsia con Aguja/estadística & datos numéricos , Boston/epidemiología , Niño , Preescolar , Criopreservación/estadística & datos numéricos , Femenino , Humanos , Estudios Longitudinales , Masculino , Persona de Mediana Edad , Variaciones Dependientes del Observador , Pruebas en el Punto de Atención/estadística & datos numéricos , Prevalencia , Radiografía Intervencional/estadística & datos numéricos , Reproducibilidad de los Resultados , Medición de Riesgo , Sensibilidad y Especificidad , Adulto Joven
16.
J Surg Oncol ; 109(6): 593-6, 2014 May.
Artículo en Inglés | MEDLINE | ID: mdl-24374823

RESUMEN

BACKGROUND AND OBJECTIVES: Local recurrence (LR) following limb-sparing surgery and radiation therapy (RT) for extremity soft tissue sarcoma (STS) is rare. The current study investigates the utility of surveillance nuclear magnetic resonance imaging (MRI) for detection of asymptomatic LRs. METHODS: The study cohort consisted of 168 adult patients with extremity STS treated with limb-sparing surgery and RT with curative intent between October 2001 and January 2011. Follow-up surveillance MRIs and history and physical examinations were performed per the NCCN guidelines with additional MRIs as clinically indicated. The method of LR detection and MRI number and indication were determined. RESULTS: After a median follow-up of 4.7 years (range: 0.6-10.5) 11 (6.5%; 11/168) patients developed LRs. Five hundred two MRIs were obtained, 429 (85.5%; 429/502) for surveillance and 73 (14.5%; 73/502) as clinically indicated. One hundred fourteen patients underwent ≥1 surveillance MRI. The median surveillance MRI interval was 6.4 months (range 1.4-68.9). Surveillance MRI detected an asymptomatic LR in 1 (0.9%; 1/114) patient with a complex reconstruction. CONCLUSIONS: Surveillance MRI infrequently detects asymptomatic LRs following limb-sparing surgery and RT for extremity STS and should be limited to patients whose primary tumor sites are not easily assessed by history and physical examination.


Asunto(s)
Extremidades/cirugía , Imagen por Resonancia Magnética , Recurrencia Local de Neoplasia/diagnóstico , Sarcoma/terapia , Neoplasias de los Tejidos Blandos/terapia , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Amputación Quirúrgica/estadística & datos numéricos , Enfermedades Asintomáticas , Quimioterapia Adyuvante , Estudios de Seguimiento , Humanos , Recuperación del Miembro , Persona de Mediana Edad , Radioterapia Adyuvante , Adulto Joven
17.
Skeletal Radiol ; 43(10): 1353-60, 2014 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-24816856

RESUMEN

OBJECTIVE: Eliglustat is an investigational oral substrate reduction therapy for Gaucher disease type 1 (GD1). Its skeletal effects were evaluated by prospective monitoring of bone mineral density (BMD), fractures, marrow infiltration by Gaucher cells, focal bone lesions, and infarcts during an open-label, multi-site, single-arm phase 2 trial (NCT00358150). MATERIALS AND METHODS: Institutional review board approval and patient informed consent were obtained. Eliglustat (50 or 100 mg) was self-administered by mouth twice daily; 19 patients completed 4 years of treatment. All were skeletally mature (age range, 18-55 years). DXA and MRI assessments were conducted at baseline and annually thereafter. X-rays were obtained annually until month 24, and then every other year. RESULTS: Lumbar spine BMD increased significantly (p = 0.02; n = 15) by a mean (SD) of 9.9% (14.2%) from baseline to year 4; corresponding T-scores increased significantly (p = 0.01) from a mean (SD) of -1.6 (1.1) to -0.9 (1.3). Mean femur T-score remained normal through 4 years. Femur MRI showed that 10/18 (56%) patients had decreased Gaucher cell infiltration compared to baseline; one patient with early improvement had transient worsening at year 4. There were no lumbar spine or femoral fractures and no reported bone crises during the study. At baseline, 8/19 (42%) patients had focal bone lesions, which remained stable, and 7/19 (37%) patients had bone infarctions, which improved in one patient by year 2. At year 4, one new asymptomatic, indeterminate bone lesion was discovered that subsequently resolved. CONCLUSIONS: Eliglustat may be a therapeutic option for treating the skeletal manifestations of GD1.


Asunto(s)
Desmineralización Ósea Patológica/tratamiento farmacológico , Densidad Ósea/efectos de los fármacos , Inhibidores Enzimáticos/uso terapéutico , Enfermedad de Gaucher/tratamiento farmacológico , Pirrolidinas/uso terapéutico , Absorciometría de Fotón/métodos , Administración Oral , Adolescente , Adulto , Desmineralización Ósea Patológica/diagnóstico , Desmineralización Ósea Patológica/etiología , Inhibidores Enzimáticos/administración & dosificación , Femenino , Fémur/diagnóstico por imagen , Fémur/efectos de los fármacos , Fémur/patología , Estudios de Seguimiento , Fracturas Óseas/etiología , Fracturas Óseas/prevención & control , Enfermedad de Gaucher/complicaciones , Humanos , Vértebras Lumbares/diagnóstico por imagen , Vértebras Lumbares/efectos de los fármacos , Vértebras Lumbares/patología , Imagen por Resonancia Magnética/métodos , Masculino , Persona de Mediana Edad , Estudios Prospectivos , Pirrolidinas/administración & dosificación , Adulto Joven
18.
Skeletal Radiol ; 42(1): 61-8, 2013 Jan.
Artículo en Inglés | MEDLINE | ID: mdl-22729377

RESUMEN

OBJECTIVE: To prospectively evaluate the incidence of delayed complications (bleeding, pain, infection) following CT-guided biopsies of bone or soft tissue lesions and to identify risk factors that predispose to their occurrence. MATERIALS AND METHODS: All adults presenting for CT-guided biopsy of a bone or soft tissue lesion were eligible for the study. Risk factors considered included patient gender and age, bone versus soft tissue, lesion location, lesion depth, anticoagulation, conscious sedation, coaxial biopsy technique, bleeding during the biopsy, dressing type and duration of placement, final diagnosis, needle gauge, number of passes, and number of days to follow-up. Outcomes measured included fever, pain, bruising/hematoma formation, and swelling and were collected by a follow-up phone call within 14 days of the biopsy. Fisher's exact test, the Wald Chi-square test, and univariate, multivariate, and stepwise logistic regression were performed to evaluate the influence of the risk factors on the outcomes. RESULTS: A total of 386 patients participated in the study. The rates of post-biopsy fever, pain, bruising, and swelling were 1.0, 16.1, 15.6, and 9.6%, respectively. Anticoagulants were identified as a risk factor for fever. Increasing patient age was identified as a risk factor for pain. Female gender and lesion location were identified as risk factors for bruising. Increasing patient age and lesion location were identified as risk factors for swelling. CONCLUSIONS: Patient age, female gender, and lesion location are risk factors for delayed minor complications following CT-guided biopsy of a bone or soft tissue lesion. There were no major complications. None of the complications in this series altered patient management.


Asunto(s)
Biopsia/métodos , Extremidades/patología , Complicaciones Posoperatorias/epidemiología , Radiografía Intervencional/métodos , Enfermedades de la Columna Vertebral/patología , Tomografía Computarizada por Rayos X , Adolescente , Adulto , Anciano , Anciano de 80 o más Años , Distribución de Chi-Cuadrado , Extremidades/diagnóstico por imagen , Femenino , Humanos , Incidencia , Modelos Logísticos , Masculino , Persona de Mediana Edad , Estudios Prospectivos , Factores de Riesgo , Enfermedades de la Columna Vertebral/diagnóstico por imagen
19.
20.
Blood ; 116(20): 4095-8, 2010 Nov 18.
Artículo en Inglés | MEDLINE | ID: mdl-20713962

RESUMEN

Eliglustat tartrate is an investigational oral substrate reduction therapy for Gaucher disease type 1 that is pharmacologically distinct from intravenous enzyme replacement therapy. Eliglustat tartrate improved clinical manifestations in patients who received 50 or 100 mg twice daily for 1 year during an open-label phase 2 study (Blood. 2010;116(6):893-899). We report further improvements after 2 years of treatment in 20 patients (11 females, 9 males; mean age, 33 years) with baseline splenomegaly and thrombocytopenia and/or anemia. Statistically significant (P < .001) percentage improvements from baseline occurred in platelet count (mean ± SD, 81% ± 56%), hemoglobin level (20% ± 15%), spleen volume (-52% ± 11%), and liver volume (-24% ± 13%). Mean platelet count increased ∼ 50 000/mm(3). Mean hemoglobin level increased 2.1 g/dL overall and 3.1 g/dL in 10 patients with baseline anemia. Organ volume reductions were greatest in patients with severe baseline organomegaly. Seventeen (85%) patients met established therapeutic goals for ≥ 3 of the 4 parameters. Lumbar spine bone mineral density increased 7.8% ± 10.6% (P = .01) and T-score 0.6 ± 0.8 (P = .012), with major gains in osteoporotic and osteopenic patients. Magnetic resonance imaging assessment showed that bone marrow infiltration by Gaucher cells was decreased (8/18 patients) or stable (10/18 patients). No safety-related trends emerged during 2 years of treatment. This multisite, open-label, single-arm phase 2 study is registered at www.clinicaltrials.gov as NCT00358150.


Asunto(s)
Huesos/patología , Inhibidores Enzimáticos/uso terapéutico , Enfermedad de Gaucher/sangre , Enfermedad de Gaucher/tratamiento farmacológico , Pirrolidinas/administración & dosificación , Pirrolidinas/uso terapéutico , Vísceras/patología , Administración Oral , Adolescente , Adulto , Huesos/efectos de los fármacos , Inhibidores Enzimáticos/administración & dosificación , Inhibidores Enzimáticos/farmacología , Femenino , Humanos , Masculino , Persona de Mediana Edad , Pirrolidinas/farmacología , Vísceras/efectos de los fármacos , Adulto Joven
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