Vascular anomaly cases for the pediatric hematologist oncologists—An interdisciplinary review

Adams, Denise M.; Brandão, Leonardo R.; Peterman, Caitlin M.; Gupta, Anita; Patel, Manish; Fishman, Steven J.; Trenor, Cameron C.

doi:10.1002/pbc.26716

Cited by 61 publications

(117 citation statements)

References 49 publications

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“…Currently, KMP is defined as profound thrombocytopenia, together with consumptive coagulopathy and hypofibrinogenemia associated only with the vascular tumors, KHE and tufted angioma (TA) ( Fig. 1) [10,11]. Conceptually, KHE and TA are part of the same neoplastic spectrum and can be present in the same biopsy specimen of the same patient [12].…”

Section: Definitionmentioning

confidence: 99%

“…Previously, KHE was shown to have an equal sex predilection. However, a slight male predominance has been indicated by two large retrospective studies, both of which collected data from more than 100 patients with KHE [10,11]. The distribution of age at onset has one peak within the first year of life when approximately 90% of KHE are evident.…”

Section: Epidemiology and Demographymentioning

confidence: 99%

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Kaposiform hemangioendothelioma: current knowledge and future perspectives

Chen

Yang

et al. 2020

Orphanet J Rare Dis

120

180

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Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm with high morbidity and mortality. The initiating mechanism during the pathogenesis of KHE has yet to be discovered. The main pathological features of KHE are abnormal angiogenesis and lymphangiogenesis. KHEs are clinically heterogeneous and may develop into a lifethreatening thrombocytopenia and consumptive coagulopathy, known as the Kasabach-Merritt phenomenon (KMP). The heterogeneity and the highly frequent occurrence of disease-related comorbidities make the management of KHE challenging. Currently, there are no medications approved by the FDA for the treatment of KHE. Multiple treatment regimens have been used with varying success, and new clinical trials are in progress. In severe patients, multiple agents with variable adjuvant therapies are given in sequence or in combination. Recent studies have demonstrated a satisfactory efficacy of sirolimus, an inhibitor of mammalian target of rapamycin, in the treatment of KHE. Novel targeted treatments based on a better understanding of the pathogenesis of KHE are needed to maximize patient outcomes and quality of life. This review summarizes the epidemiology, etiology, pathophysiology, clinical features, diagnosis and treatments of KHE. Recent new concepts and future perspectives for KHE will also be discussed.

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Section: Definitionmentioning

confidence: 99%

Section: Epidemiology and Demographymentioning

confidence: 99%

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Chen

Yang

et al. 2020

Orphanet J Rare Dis

120

180

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“…GLA and GSD are distinct entities with bone involvement . The clinical and radiological features of the two disorders have some specific and some overlapping features, but regardless of the final diagnosis, any bone destruction associated with LM mandates full evaluation for CLA …”

Section: Recommendationsmentioning

confidence: 99%

Multidisciplinary guidelines for initial evaluation of complicated lymphatic anomalies—expert opinion consensus

Iacobas

Adams

Pimpalwar

et al. 2019

Pediatric Blood & Cancer

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Objective Complicated lymphatic anomalies (CLAs) are chronic, progressive, and debilitating conditions that share clinical features, yet key elements for optimal evaluation and management have not been established. We aimed to formulate expert opinion consensus‐based guidelines for comprehensive evaluation of CLAs. Study Design Patient support groups dedicated to CLAs organized an international conference for vascular anomaly experts from 16 specialties to address the objective. Participants received a set of questions before the meeting and reviewed the literature. Data extracted from international lymphatic anomaly registries were presented and the group separated for panel discussions during the conference. The recommendations achieving consensus within the panel were presented to the entire audience. Open debate occurred until majority approval was achieved. Results The expert group was composed of 52 physicians who defined the clinical elements required to evaluate and diagnose a CLA. The radiology panel established the preferred anatomical and functional imaging methods for diagnosis and the elements required to be described during interpretation. Two medical panels compiled the metabolic and hematologic tests at diagnosis and also recommended functional studies. The surgical group recommended precautions for biopsy and the pathology panel provided biopsy specimen processing guidelines. Conclusions Patients with CLAs require a comprehensive and targeted diagnostic plan for appropriate management, prevention of complications, and conservation of resources. As this population is managed by diverse medical and surgical specialties, we offer an expert multidisciplinary consensus‐based opinion on the current literature and on data extracted from international lymphatic anomaly registries.

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“…The recent discovery of several lymphatic endothelial molecular markers, such as Prox‐1, podoplanin, and vascular endothelial growth factor receptor 3 (VEGFR3), has enabled the detection of lymphatic endothelial cells in affected lesions. However, open biopsy risks severe complications including bleeding, infection, and refractory effusions, especially rib biopsy . Therefore, the development of noninvasive and alternative methods and biomarkers is essential for LA patients and clinicians.…”

Section: Introductionmentioning

confidence: 99%

“…However, open biopsy risks severe complications including bleeding, infection, and refractory effusions, especially rib biopsy. 6 Therefore, the development of noninvasive and alternative methods and biomarkers is essential for LA patients and clinicians.…”

Section: Introductionmentioning

confidence: 99%

Potential biomarkers of kaposiform lymphangiomatosis

Ozeki

Nozawa

Kawamoto

et al. 2019

Pediatric Blood & Cancer

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Background Kaposiform lymphangiomatosis (KLA) has recently been distinguished as a novel subtype of generalized lymphatic anomaly (GLA), and is characterized by foci of spindle endothelial cells amid a background of malformed lymphatic channels. The etiology of these diseases remains unknown and diagnosis is confounded by their similar clinical findings. This study aimed to clarify differences in the clinical findings and plasma cytokine profiles of GLA and KLA patients. Procedure Clinical features data of GLA and KLA patients were obtained from a national survey. Differences in clinical findings, plasma levels of cytokines, and survival were analyzed. Plasma was obtained from healthy controls and GLA and KLA patients. Thirty‐six angiogenic and lymphangiogenic factors were evaluated for cytokine concentration. Results Twenty‐one patients with GLA and 11 with KLA were recruited. Mediastinal masses, hemorrhagic pericardial and pleural effusion, coagulation disorders, and thrombocytopenia were more frequent in KLA than in GLA. KLA had a significantly poorer outcome than GLA (P = 0.044). Soluble VEGFR3, angiopoietin 2, HGF, soluble HER2, tenascin C, and soluble HGFR levels were higher in KLA. Notably, soluble VEGFR3 and angiopoietin 2 levels were approximately 10‐fold higher than those of other molecules measured. However, soluble VEGFR1 and soluble TIE2 were lower in KLA than in GLA and the controls. Conclusions Patients with KLA have an unfavorable prognosis and serious symptoms (hemorrhagic pleural effusion and coagulation disorders). Our data indicate that eight angiogenic cytokines might be potential biomarkers of KLA.

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Vascular anomaly cases for the pediatric hematologist oncologists—An interdisciplinary review

Cited by 61 publications

References 49 publications

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Kaposiform hemangioendothelioma: current knowledge and future perspectives

Multidisciplinary guidelines for initial evaluation of complicated lymphatic anomalies—expert opinion consensus

Potential biomarkers of kaposiform lymphangiomatosis

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