Accuracy of ultrasonography in assessing spleen and liver size in patients with Gaucher disease: comparison to computed tomographic measurements

Elstein, Deborah; Hadas‐Halpern, Irith; Azuri, Y; Abrahamov, Ayala; Bar-Ziv, Y; Zimran, Ari

doi:10.7863/jum.1997.16.3.209

Cited by 68 publications

(60 citation statements)

References 3 publications

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“…Ultrasound measurements are converted to volumetric equivalents using the formula of Elstein et al [34]. Registry procedures recommend technological consistency for serial volumetric measurements, but compliance was not verified for each patient.…”

Section: Therapeutic Goalsmentioning

confidence: 99%

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

Weinreb¹,

Taylor²,

Cox

et al. 2008

American J Hematol

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To assess the extent to which patients with type 1 Gaucher disease (GD1) receiving individualized enzyme replacement therapy with imiglucerase attain six defined therapeutic goals. One hundred and ninety-five GD1 patients enrolled in the ICGG Gaucher Registry, all of whom had data available for hemoglobin, platelet count, liver volume, spleen volume, bone pain, and bone crises at first infusion and after 4 years of therapy with imiglucerase, were evaluated for achievement of published therapeutic goals. The proportion of patients who met all six therapeutic goals increased from 2.1% at first infusion to 41.5% at 4 years; ‡5 goals from 12.8% to 76.9%; ‡4 goals from 37.4% to 92.8%; ‡3 goals from 70.8% to 99.0%; and ‡2 goals from 95.4% to 99.5%. All patients met at least one goal at first infusion and after 4 years of treatment. The proportion of patients meeting specific therapeutic goals increased for all parameters between first infusion and 4 years of therapy: platelet count (24.6%-79.5%), spleen volume (25.6%-78.5%), liver volume (45.6%-90.8%), bone pain (62.6-70.3%), hemoglobin (68.2-91.8%), and bone crises (91.8-99.0%). On average, patients who received higher doses of imiglucerase achieved a greater number of therapeutic goals. This analysis provides a benchmark for evaluating the utility of a disease management approach for GD1 based on monitoring achievement of therapeutic goals after treatment with imiglucerase. Am. J. Hematol. 83:890-895, 2008. V

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Section: Therapeutic Goalsmentioning

confidence: 99%

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

Weinreb¹,

Taylor²,

Cox

et al. 2008

American J Hematol

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“…My experience with ultrasound scanning for routine follow-up of organ volumes has been rewarding despite reliance on a single radiologist 31 ; for specific indications and for clinical trial assessments, magnetic resonance imaging is preferable.…”

Section: Imaging Modalitiesmentioning

confidence: 99%

How I treat Gaucher disease

Zimran

2011

Blood

145

135

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This review presents a cohesive approach to treating patients with Gaucher disease. The spectrum of the clinical presentation of the disease is broad, yet heretofore there was only one diseasespecific treatment. In the past 2 years, a global shortage of this product has resulted in reassessment of the "one enzyme-one disease-one therapy" mantra. It has also showcased the multiple levels that engage the patient, the treating physician, and the third-party insurer in providing adequate treatment to all symptomatic patients. The key points summarizing the way I manage my patients include accurate enzymatic diagnosis with mutation analysis (for some prognostication and better carrier detection in the family), a detailed follow-up every 6-12 months (with an option to see consultants and attention to comorbidities), and initiation of enzyme replacement therapy according to symptoms or deterioration in clinically significant features or both. I do not treat patients with very mild disease, but I consider presymptomatic therapy for patients at risk, including young women with poor obstetric history. I prefer the minimal-effective dose rather than the maximally tolerated dose, and when the difference between high-dose and lower-dose regimens is (merely statistically significant but) clinically meaningless, minimizing the burden on society by advocating less-expensive treatments is ethically justified. (Blood. 2011;118(6): 1463-1471) IntroductionThe purpose of this review is to present a cohesive approach to treating patients with Gaucher disease (GD). As can be appreciated, the spectrum of the clinical presentation of the disease is broad, yet heretofore there was only one disease-specific treatment. In the past 2 years, a global shortage of this product has resulted in reassessment of the "one enzyme-one disease-one therapy" mantra. It has also showcased the multiple levels that engage the patient, the treating physician, and the third-party insurer, in providing adequate treatment for all symptomatic patients with GD. As a treating physician dedicated to GD even before the era of enzyme replacement therapy (ERT), my mandate and inspiration has always been to provide the best therapy with the least malfeasance. Description of GDGD is one of the most common glycolipid storage disorders, caused by an inherited deficiency of the lysosomal enzyme ␤-glucocerebrosidase, leading to accumulation of the substrate glucocerebroside in the cells of the macrophage-monocyte system. 1 Accordingly, key disease features are related to splenomegaly with hypersplenism, hepatomegaly, and bone involvement. Although a single-gene disorder, phenotypic expression is extremely variable, ranging from totally asymptomatic (only detectable by DNA analysis or enzyme deficiency) to a lethal newborn form with hydrops fetalis and ichthyosis. Many manifestations, some notuncommon and others very rare, cannot be explained by storage per se; examples include immunologic abnormalities, increased prevalence of certain malignancies with relative paucity of ...

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“…[1][2][3][4][5][6][7] In addition to diagnosis, organ volume/height measurements have also been found to be important in making surgical decisions involving organ transplantation. [8][9][10] In traditional clinical practice, 3D organ analysis is performed via time-consuming manual measurements; alternatively, the evaluation is based on 2D projection images, which introduces bias.…”

Section: Introductionmentioning

confidence: 99%

Automated segmentation and quantification of liver and spleen from CT images using normalized probabilistic atlases and enhancement estimation

et al. 2010

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Purpose:To investigate the potential of the normalized probabilistic atlases and computer-aided medical image analysis to automatically segment and quantify livers and spleens for extracting imaging biomarkers ͑volume and height͒. Methods: A clinical tool was developed to segment livers and spleen from 257 abdominal contrastenhanced CT studies. There were 51 normal livers, 44 normal spleens, 128 splenomegaly, 59 hepatomegaly, and 23 partial hepatectomy cases. 20 more contrast-enhanced CT scans from a public site with manual segmentations of mainly pathological livers were used to test the method. Data were acquired on a variety of scanners from different manufacturers and at varying resolution. Probabilistic atlases of livers and spleens were created using manually segmented data from ten noncontrast CT scans ͑five male and five female͒. The organ locations were modeled in the physical space and normalized to the position of an anatomical landmark, the xiphoid. The construction and exploitation of liver and spleen atlases enabled the automated quantifications of liver/spleen volumes and heights ͑midhepatic liver height and cephalocaudal spleen height͒ from abdominal CT data. The quantification was improved incrementally by a geodesic active contour, patient specific contrast-enhancement characteristics passed to an adaptive convolution, and correction for shape and location errors. Results: The livers and spleens were robustly segmented from normal and pathological cases. For the liver, the Dice/Tanimoto volume overlaps were 96.2%/92.7%, the volume/height errors were 2.2%/2.8%, the root-mean-squared error ͑RMSE͒ was 2.3 mm, and the average surface distance ͑ASD͒ was 1.2 mm. The spleen quantification led to 95.2%/91% Dice/Tanimoto overlaps, 3.3%/ 1.7% volume/height errors, 1.1 mm RMSE, and 0.7 ASD. The correlations ͑R 2 ͒ with clinical/ manual height measurements were 0.97 and 0.93 for the spleen and liver, respectively ͑p Ͻ 0.0001͒. No significant difference ͑p Ͼ 0.2͒ was found comparing interobserver and automaticmanual volume/height errors for liver and spleen. Conclusions: The algorithm is robust to segmenting normal and enlarged spleens and livers, and in the presence of tumors and large morphological changes due to partial hepatectomy. Imaging biomarkers of the liver and spleen from automated computer-assisted tools have the potential to assist the diagnosis of abdominal disorders from routine analysis of clinical data and guide clinical management.

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Accuracy of ultrasonography in assessing spleen and liver size in patients with Gaucher disease: comparison to computed tomographic measurements

Cited by 68 publications

References 3 publications

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

A benchmark analysis of the achievement of therapeutic goals for type 1 Gaucher disease patients treated with imiglucerase

How I treat Gaucher disease

Automated segmentation and quantification of liver and spleen from CT images using normalized probabilistic atlases and enhancement estimation

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