Browse
Submit Data
Databases
API
Help

Dataset Information

0 Views

0 Connections

0 Citations

0 Reanalyses

0 Downloads

Omics score: 0

Case report: Experience of a rare case of rebound of the Kasabach-Merritt phenomenon during sirolimus treatment in kaposiform hemangioendothelioma.

ABSTRACT: Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm associated with the Kasabach-Merritt phenomenon (KMP), which is a consumptive coagulopathy with associated potentially life-threatening thrombocytopenia. There are no standardized treatment protocols for the management of KHE with KMP. Moreover, there are limited reports regarding the treatment of cases of rebound. Herein, we describe a rare case of rebound of KHE/KMP, during systemic sirolimus treatment, successfully treated with embolization and vincristine infusion combined with microwave ablation.

SUBMITTER: Wang L

PROVIDER: S-EPMC9391052 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature

ACCESS DATA

Json Xml

Publications

Case report: Experience of a rare case of rebound of the Kasabach-Merritt phenomenon during sirolimus treatment in kaposiform hemangioendothelioma.

Wang Liang L Li Jing J Wu Changhua C Song Dan D Liu Zhuang Z Niu Yanli Y Zhou Jie J Guo Lei L

Frontiers in pediatrics 20220805

Kaposiform hemangioendothelioma (KHE) is a rare vascular neoplasm associated with the Kasabach-Merritt phenomenon (KMP), which is a consumptive coagulopathy with associated potentially life-threatening thrombocytopenia. There are no standardized treatment protocols for the management of KHE with KMP. Moreover, there are limited reports regarding the treatment of cases of rebound. Herein, we describe a rare case of rebound of KHE/KMP, during systemic sirolimus treatment, successfully treated with ...[more]

PMID: 35990005

Similar Datasets

Platelet functional abnormalities in pediatric patients with kaposiform hemangioendothelioma/Kasabach-Merritt phenomenon.

Project description:Kaposiform hemangioendothelioma (KHE) is a rare vascular tumor of infancy that is commonly associated with a life-threatening thrombocytopenic condition, Kasabach-Merritt phenomenon (KMP). Platelet CLEC-2, tumor podoplanin interaction is considered the key mechanism of platelet clearance in these patients. Here, we aimed to assess platelet functionality in such patients. Three groups of 6 to 9 children were enrolled: group A with KHE/KMP without hematologic response (HR) to therapy; group B with KHE/KMP with HR; and group C with healthy children. Platelet functionality was assessed by continuous and end point flow cytometry, low-angle light scattering analysis (LaSca), fluorescent microscopy of blood smears, and ex vivo thrombi formation. Platelet integrin activation in response to a combination of CRP (GPVI agonist) and TRAP-6 (PAR1 agonist), as well as calcium mobilization and integrin activation in response to CRP or rhodocytin (CLEC-2 agonist) alone, were significantly diminished in groups A and B. At the same time, platelet responses to ADP with or without TRAP-6 were unaltered. Thrombi formation from collagen in parallel plate flow chambers was also noticeably decreased in groups A and B. In silico analysis of these results predicted diminished amounts of CLEC-2 on the platelet surface of patients, which was further confirmed by immunofluorescence microscopy and flow cytometry. In addition, we also noted a decrease in GPVI levels on platelets from group A. In KHE/KMP, platelet responses induced by CLEC-2 or GPVI activation are impaired because of the diminished number of receptors on the platelet surface. This impairment correlates with the severity of the disease and resolves as the patient recovers.

| S-EPMC10463204 | biostudies-literature

Pancreatic kaposiform hemangioendothelioma presenting with duodenal obstruction and kasabach-merritt phenomenon: a neonate cured by whipple operation.

Project description:Aim Kaposiform hemangiondothelioma (KHE) is a rare vascular tumor, commonly associated with Kasaback-Merritt phenomenon characterized by thrombocytopenia and consumptive coagulopathy. We report a case of pancreatic KHE presenting with neonatal duodenal obstruction and Kasaback-Merritt phenomenon. Case Report A full term male baby presented with bile stained vomiting on Day 3 of life. Contrast study and computed tomography scan showed duodenal obstruction by a 5 cm extrinsic hypervascular mass. Platelet count was 23 x 109/L. Laparotomy confirmed a vascular tumor arising from the pancreatic head compressing on the duodenum. Whipple operation was performed. Results Intestinal obstruction and thrombocytopenia resolved after surgery. There was no post-operative complications. Histology confirmed KHE. The boy was tolerating hydrolyzed milk formula and was thriving at 5 months follow up. Conclusion We reported a case of pancreatic KHE presented with neonatal intestinal obstruction and Kasaback-Merritt phenomenon. High index of suspicion is necessary for diagnosis. To our knowledge, this is the youngest patient who underwent Whipple operation.

| S-EPMC4336109 | biostudies-other

Impact of age and tumor size on the development of the Kasabach–Merritt phenomenon in patients with kaposiform hemangioendothelioma: a retrospective cohort study

Project description:Abstract Introduction The Kasabach–Merritt phenomenon (KMP) is a severe complication of kaposiform hemangioendothelioma (KHE). The risk factors for KMP need further investigation. Methods The medical records of patients with KHE were reviewed. Univariate and multivariate logistic regression models were used for the risk factors for KMP, and the area under the receiver operator characteristic (ROC) curve was used to assess the predictive power of risk factors. Results A total of 338 patients with KHE were enrolled. The incidence of KMP was 45.9%. Age of onset (P < 0.001, odds ratio [OR] 0.939; 95% confidence interval [CI] 0.914–0.966), lesion size (P < 0.001, OR 1.944; 95% CI 1.646–2.296), mixed type (P = 0.030, OR 2.428; 95% CI 1.092–5.397), deep type (P = 0.010, OR 4.006; 95% CI 1.389–11.556), and mediastinal or retroperitoneal lesion location (P = 0.019, OR 11.864; 95% CI 1.497–94.003) were correlated with KMP occurrence through multivariate logistic regression. ROC curve analysis revealed that the optimal cutoffs were 4.75 months for the age of onset (P < 0.001, OR 7.206, 95% CI 4.073–12.749) and a lesion diameter of 5.35 cm (P < 0.001, OR 11.817, 95% CI 7.084–19.714). Bounded by a lesion size of 5.35 cm, we found significant differences in tumor morphology, age of onset, treatments, and hematological parameters. Using an onset age of 4.75 months as a cutoff, we found significant differences in tumor morphology, lesion size, hematological parameters, and prognosis. Conclusion For KHE patients with an onset age <4.75 months and/or lesion diameter >5.35 cm, clinicians should be wary of the occurrence of KMP. Active management is recommended to improve the prognosis.

| S-EPMC10249050 | biostudies-literature

Case Report: Kaposiform hemangioendothelioma with PIK3CA mutation successfully treated with sirolimus

Project description:Kaposiform hemangioendothelioma (KHE) is an extremely rare, locally aggressive vascular neoplasm. The etiopathogenesis of KHE is still poorly understood. In the present study, we found a new mutation in KHE (c.685delA, p.Thr229fs). The KHE patient with the PIK3CA mutation showed complete regression after sirolimus treatment. We propose that the presence of the PIK3CA mutation in KHE may correlate with good response to sirolimus.

| S-EPMC10235594 | biostudies-literature

Two cases of hemangioma with Kasabach-Merritt phenomenon diagnosed via ultrasound

Project description: Not available

| S-EPMC10498253 | biostudies-literature

Management of pancreatic kaposiform hemangioendothelioma with sirolimus in a pediatric patient: a case report and literature review.

Project description:BackgroundKaposiform hemangioendothelioma (KHE) is a rare locally aggressive vascular tumor. The pancreas is not a common site of KHE, especially in pediatric patients. Given that no guidelines are available for the treatment of KHE, management is currently based on expert opinions and clinical experiences. Here, we report a case of pancreatic KHE with obstructive jaundice, which was treated successfully with oral sirolimus instead of radical surgery. Additionally, a literature review on pancreatic KHE was performed to summarize prior clinical experiences and the available treatments.Case descriptionA 10-month-old Chinese male infant presented with obstructive jaundice without any signs of fever, abdominal pain, or distention. A detailed consultation revealed an uneventful history. The obstructive jaundice worsened significantly during 3 weeks of conservative therapy. A pancreatic mass was identified via radiological evidence, and a laparoscopic biopsy of the tumor was performed, which confirmed the diagnosis of pancreatic KHE based on histological findings. Oral sirolimus 0.8 mg/m2 twice daily was administered at a steady serum concentration of 5-15 ng/mL, which led to a shrinkage in tumor size and resolution of jaundice. The patient showed no evidence of recurrence after 1 year of follow-up and is still on sirolimus treatment, which has been tolerated well up to the time of this report.ConclusionsThe pancreas is a rare location of KHE, which is a locally aggressive vascular tumor. Diagnosis is based on histological findings, and therapy should be multidisciplinary and individualized. Although sirolimus has been very successful in the treatment of KHE even without radical surgery, the possible risks of tumor recurrence and adverse effects warrant some caution.

| S-EPMC9442215 | biostudies-literature

Population pharmacokinetics of sirolimus in pediatric patients with kaposiform hemangioendothelioma: A retrospective study.

Project description:Numerous studies have established population pharmacokinetics (PPK) models of sirolimus in various populations. However, a PPK model of sirolimus in Chinese patients with pediatric kaposiform hemangioendothelioma (PKHE) has yet to be established; therefore, this was the purpose of the present study. The present study was a retrospective analysis that utilized the trough concentration data obtained from traditional therapeutic drug monitoring-based dose adjustments. A total of 17 Chinese patients with PKHE from a real-world study were characterized by non-linear mixed-effects modeling. The impact of demographic features, biological characteristics and concomitant medications was assessed. The developed final model was evaluated via bootstrap and a prediction-corrected visual predictive check. A one-compartment model with first-order absorption and elimination was used for modeling of data for PKHE. The typical values of apparent oral clearance (CL/F) and apparent volume of distribution (V/F) in the final model were 3.19 l/h and 165 liters, respectively. Age, alanine transaminase levels and sex were included as significant covariates for CL/F, while the duration of treatment with sirolimus was a significant covariate for V/F. In conclusion, the present study developed and validated the first sirolimus PPK model for Chinese patients with PKHE.

| S-EPMC6676569 | biostudies-literature

Kaposiform lymphangiomatosis and kaposiform hemangioendothelioma: similarities and differences.

Project description:BackgroundKaposiform lymphangiomatosis (KLA) and kaposiform hemangioendothelioma (KHE) are rare and aggressive vascular disorders. The aim of this study was to examine the clinical features and prognosis of KLA and KHE involving the thorax.MethodsThe clinical features, imaging and pathological findings, treatments and outcome were retrospectively reviewed for 6 patients with KLA and 7 patients with KHE involving the thorax.ResultsThe mean ages at the time of the presentation of signs/symptoms were 26.7 months and 4.1 months for KLA and KHE, respectively. Respiratory symptoms, pericardial and pleural effusion, thrombocytopenia and coagulopathy were common in both KLA and KHE. Diffuse lesions involving the lung and extrathoracic sites were observed in KLA but not in KHE. Histopathologically, all lesions had spindled tumor cells, which were immunoreactive for CD31 and D2-40. In KLA, the spindle cells were distributed in sparse and poorly marginated clusters, whereas the spindle cells formed more defined and confluent vascularized nodules in KHE. Unlike the refractory behavior of KLA, the majority of patients with KHE responded to medical treatments with regression of the lesion and normalization of the hematologic parameters.ConclusionsThe presenting and histological characteristics of KLA can overlap with those of KHE. The presence of diffuse vascular lesions in the mediastinum and lung with refractory thrombocytopenia and coagulopathy should suggest the diagnosis of KLA. Given the rarity and high morbidity and mortality of these disorders, the diagnostic process and therapeutic approach should include a multidisciplinary team review and consensus.

| S-EPMC6612206 | biostudies-literature

Case Report: Congenital Intracranial Kaposiform Hemangioendothelioma Treated With Surgical Resection.

Project description:BackgroundKaposiform hemangioendothelioma (KHE) is a locally aggressive but non-metastatic vascular neoplasm. Most studies have been restricted to small case series of limited generalizability. Intracranial KHE is extremely rare with only three cases reported in the literature. Here, we report a case of congenital intracranial KHE who underwent surgical resection, and no lesion recurrence was seen during the follow-up period of 13 months.Case descriptionA 2-month-old boy initially presented with a left temporal mass following birth. Antenatal ultrasound at 36 weeks of gestation demonstrated a hyperechoic signal present in the left frontal lobe, with clear borders and irregular morphology. There were neither cutaneous abnormalities nor other neurologic examination abnormalities. No laboratory abnormality was identified. Computed tomography (CT) scans suggested that a massive hematoma was noted under the left frontal skull plate, with a little subdural hemorrhage in the adjacent temporal area. The adjacent meninges enhanced and thickened on contrasted T1 magnetic resonance (MR). After the multidisciplinary diagnostic assessment, the surgery was performed by the left frontotemporal craniotomy approach. The operation was extremely difficult. We completely removed the tumor, and the involved dura and brain tissue were resected with the lesion in a piecemeal fashion. On postoperative-day (POD) 3 and POD 14, the head CT re-examination revealed that cerebral perfusion improved gradually. The MRI of 6- and 12-month after operation showed no local recurrence or metastasis.ConclusionsIntracranial KHE is difficult to diagnose early and the prognosis has been uniformly poor. We supposed that meticulous intraoperative hemostasis is the key for a successful operation, and the radical resection of the tumor and involved structures are essential to reduce recurrence.

| S-EPMC9012330 | biostudies-literature

Kaposiform hemangioendothelioma: current knowledge and future perspectives.

Project description: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 life-threatening 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.

| S-EPMC6998257 | biostudies-literature

OmicsDI is part of the ELIXIR infrastructure

OmicsDI is an Elixir interoperability service. Learn more ›

Tweets

OmicsDI Databases

PRIDE
PeptideAtlas
MassIVE
JPOST Repository
Physiome Model Repository

EGA
EVA
ENA
LINCS
PAXDB
Cell Collective

MetaboLights
Metabolomics Workbench
MetabolomeExpress
GNPS
BioModels
FAIRDOMHub

ArrayExpress
dbGaP
ExpressionAtlas
GEO
NODE

Information

Databases
Help
API
Contact us
Code on GitHub
Terms of use
Submit Data