scholarly journals Sirolimus in the treatment of kaposiform lymphangiomatosis

2021 ◽  
Vol 16 (1) ◽  
Author(s):  
Jiangyuan Zhou ◽  
Kaiying Yang ◽  
Siyuan Chen ◽  
Yi Ji
Keyword(s):  
Clinical Practice ◽  
Poor Prognosis ◽  
Standard Treatment ◽  
Mammalian Target Of Rapamycin ◽  
Refractory Disease ◽  
Generalized Lymphatic Anomaly ◽  
The Poor ◽  
Poor Understanding ◽  
Kaposiform Lymphangiomatosis ◽  
New Treatments

Abstract Background Kaposiform lymphangiomatosis (KLA), which is a new subtype of generalized lymphatic anomaly, is a rare disease with a poor prognosis. Currently, there is no standard treatment due to the poor understanding of KLA. Sirolimus, which is an inhibitor of mammalian target of rapamycin, has been shown to have promising potential in the treatment of complicated vascular anomalies. The aim of this study was to introduce the use of sirolimus for the treatment of KLA and to highlight the challenges of managing this refractory disease. Results We reported seven patients with KLA who received sirolimus therapy in our center. Combined with previously reported cases, 58.3% achieved a partial response, 25.0% had stable disease, and 16.7% experienced disease progression. No severe sirolimus-related adverse events occurred during treatment. Conclusions This study suggests that sirolimus is currently an option for the treatment of KLA, and it is hoped that more specific therapies will be developed in the future. Rapid advances in basic science and clinical practice may facilitate the development of important new treatments for KLA.

scholarly journals A Case of Suspected Adverse Reactions to Sirolimus in the Treatment of Generalized Lymphatic Anomaly

2019 ◽  
Vol 2019 ◽  
pp. 1-4
Author(s):  
Takayuki Fujii ◽  
Ryuichi Shimono ◽  
Aya Tanaka ◽  
Hiroto Katami
Keyword(s):  
Pleural Effusion ◽  
Disseminated Intravascular Coagulation ◽  
Poor Prognosis ◽  
Adverse Reactions ◽  
Standard Treatment ◽  
Mammalian Target Of Rapamycin ◽  
Lymphatic Vessels ◽  
Drug Reactions ◽  
Generalized Lymphatic Anomaly ◽  
Suspected Adverse Reactions

Generalized lymphatic anomaly (GLA) is characterized by diffuse or multicentric proliferation of dilated lymphatic vessels resembling common lymphatic malformation, and thoracic lesions can be related to a poor prognosis. Sirolimus, an inhibitor of the mammalian target of rapamycin, is effective against vascular anomalies with few severe adverse drug reactions. Here, we report the case of a patient with intractable hemothorax pleural effusion due to GLA who was treated with sirolimus and experienced disseminated intravascular coagulation. Although a standard treatment for GLA has not been established, pleural fluid might be reduced using the Kampo medicine Eppikajyutsuto.

scholarly journals The Effect of Heterogenous Subregions in Glioblastomas on Survival Stratification: A Radiomics Analysis Using the Multimodality MRI

2021 ◽  
Vol 20 ◽  
pp. 153303382110330
Author(s):  
Lulu Yin ◽  
Yan Liu ◽  
Xi Zhang ◽  
Hongbing Lu ◽  
Yang Liu
Keyword(s):  
Poor Prognosis ◽  
Intratumor Heterogeneity ◽  
Short Term ◽  
Prognostic Information ◽  
Manual Delineation ◽  
Flair Sequence ◽  
The Poor ◽  
Contrast Enhanced ◽  
Mri Sequences

Intratumor heterogeneity is partly responsible for the poor prognosis of glioblastoma (GBM) patients. In this study, we aimed to assess the effect of different heterogeneous subregions of GBM on overall survival (OS) stratification. A total of 105 GBM patients were retrospectively enrolled and divided into long-term and short-term OS groups. Four MRI sequences, including contrast-enhanced T1-weighted imaging (T1C), T1, T2, and FLAIR, were collected for each patient. Then, 4 heterogeneous subregions, i.e. the region of entire abnormality (rEA), the regions of contrast-enhanced tumor (rCET), necrosis (rNec) and edema/non-contrast-enhanced tumor (rE/nCET), were manually drawn from the 4 MRI sequences. For each subregion, 50 radiomics features were extracted. The stratification performance of 4 heterogeneous subregions, as well as the performances of 4 MRI sequences, was evaluated both alone and in combination. Our results showed that rEA was superior in stratifying long-and short-term OS. For the 4 MRI sequences used in this study, the FLAIR sequence demonstrated the best performance of survival stratification based on the manual delineation of heterogeneous subregions. Our results suggest that heterogeneous subregions of GBMs contain different prognostic information, which should be considered when investigating survival stratification in patients with GBM.

scholarly journals Umbilical cord blood transplantation can overcome the poor prognosis of KMT2A-MLLT3 acute myeloid leukemia and can lead to good GVHD-free/relapse-free survival

2021 ◽  
Author(s):  
Juan Tong ◽  
Lei Zhang ◽  
Huilan Liu ◽  
Xiucai Xu ◽  
Changcheng Zheng ◽  
...  
Keyword(s):  
Myeloid Leukemia ◽  
Poor Prognosis ◽  
Cord Blood Transplantation ◽  
Chronic Gvhd ◽  
Relapse Free Survival ◽  
Free Survival ◽  
Chemotherapy Group ◽  
Blood Transplantation ◽  
The Poor ◽  
First Complete Remission

AbstractThis is a retrospective study comparing the effectiveness of umbilical cord blood transplantation (UCBT) and chemotherapy for patients in the first complete remission period for acute myeloid leukemia with KMT2A-MLLT3 rearrangements. A total of 22 patients were included, all of whom achieved first complete remission (CR1) through 1–2 rounds of induction chemotherapy, excluding patients with an early relapse. Twelve patients were treated with UCBT, and 10 patients were treated with chemotherapy after 2 to 4 courses of consolidation therapy. The 3-year overall survival (OS) of the UCBT group was 71.3% (95% CI, 34.4–89.8%), and that of the chemotherapy group was 10% (95% CI, 5.89–37.3%). The OS of the UCBT group was significantly higher than that of the chemotherapy group (P = 0.003). The disease-free survival (DFS) of the UCBT group was 60.8% (95% CI, 25.0–83.6%), which was significantly higher than the 10% (95% CI, 5.72–35.8%) of the chemotherapy group (P = 0.003). The relapse rate of the UCBT group was 23.6% (95% CI, 0–46.8%), and that of the chemotherapy group was 85.4% (95% CI, 35.8–98.4%), which was significantly higher than that of the UCBT group (P < 0.001). The non-relapse mortality (NRM) rate in the UCBT group was 19.8% (95% CI, 0–41.3%), and that in the chemotherapy group was 0.0%. The NRM rate in the UCBT group was higher than that in the chemotherapy group, but there was no significant difference between the two groups (P = 0.272). Two patients in the UCBT group relapsed, two died of acute and chronic GVHD, and one patient developed chronic GVHD 140 days after UCBT and is still alive, so the GVHD-free/relapse-free survival (GRFS) was 50% (95% CI, 17.2–76.1%). AML patients with KMT2A-MLLT3 rearrangements who receive chemotherapy as their consolidation therapy after CR1 have a very poor prognosis. UCBT can overcome the poor prognosis and significantly improve survival, and the GRFS for these patients is very good. We suggest that UCBT is a better choice than chemotherapy for KMT2A-MLLT3 patients.

scholarly journals AB0882-HPR ANCA-ASSOCIATED VASCULITIS: A CLINICAL PRACTICE ASSESSMENT

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 1464.2-1465
Author(s):  
S. Mendly ◽  
G. Boutsalis
Keyword(s):  
Clinical Practice ◽  
Clinical Trial Data ◽  
Trial Data ◽  
Remission Induction ◽  
Refractory Disease ◽  
Maintenance Strategy ◽  
Combination Therapies ◽  
Research Support ◽  
Educational Grant ◽  
Anca Associated Vasculitis

Background:AAV (ANCA-associated vasculitis) is a group of progressive, rare, severe autoimmune diseases1,2. AAV can affect blood vessels in different parts of the body resulting in damage to vital organs such as the lungs, kidneys, nervous system, gastrointestinal system, skin, eyes, and heart.2 There are currently no approved therapies for remission-induction in patients with AAV. The current treatment armamentarium for AAV is comprised of various immunosuppressive therapies in combination with steroid treatment. Understanding clinical practice gaps in the management of AAV, can inform development of tools to improve physician practices.Objectives:This medical education activity aims to assess physicians’ knowledge on the various manifestations of antineutrophil cytoplasmic antibody-associated vasculitis (AAV), current guideline-recommended treatment strategies for remission induction in patients with AAV, as well as recent clinical trial data for combination therapies used for remission induction.Methods:A 24-question survey consisting of multiple-choice knowledge and case-based questions was made available to nephrologists and rheumatologists without monetary compensation or charge. The questions were designed to evaluate knowledge regarding the various manifestations of AAV and the results from clinical trials that have compared the efficacy of combination therapies used for remission induction in patients with AAV. As well as application of guideline-recommended therapies and clinical trial data for remission induction in patients with AAV within clinical practice. The survey launched online on a website dedicated to continuous professional development. (www.medscape.org/viewarticle/920320) on July 15, 2020. Data were collected until October 1, 2020.Results:363 nephrologists and 190 rheumatologists completed the survey. Physicians demonstrated gaps in the following areas:TopicIncorrect Responses to Knowledge and Clinical Decision-Making Questions (%)NephrologistsRheumatologistsSystemic diseases associated with AAV59%45%How to confirm diagnosis of AAV42%25%Therapy selection to induce remission that would be consistent with guidelines recommendations71%51%Guideline-recommended therapy for patients that do not respond to the induction regimen32%34%Definition of refractory disease95%94%Most effective maintenance strategy for a patient once remission is achieved80%64%Where would an emerging therapy such as a C5a receptor inhibitor fit into the therapeutic armamentarium of AAV?62%47%What are the guideline-recommended therapies to reduce remission in patients without organ-threatening disease?71%51%Most effective maintenance strategy for a patient once remission is achieved80%64%Guideline-recommendations on length of time to continue maintenance therapy31%35%Conclusion:This educational research on assessment of physicians’ (nephrologists and rheumatologists) clinical practices yielded important insights into clinical gaps related to understanding of the disease pathophysiology and progression of AAV, guideline recommendations on diagnosing and managing AAV with guideline-directed medical therapies (GDMTs), strategies for the management of relapsing and refractory disease in AAV and positioning of emerging therapies in the treatment paradigm.References:[1]www.medscape.org/viewarticle/920320.[2]Hutton HL, et al. Semin Nephrol 2017;37(5):418–35.[3]Al-Hussain T, et al. Adv Anat Pathol 2017;24(4):226–34.Disclosure of Interests:Sarah Mendly Grant/research support from: Supported by an independent educational grant from Vifor Pharma, George Boutsalis Grant/research support from: Supported by an independent educational grant from Vifor Pharma

Ionic liquids: Solvation ability and polarity

2009 ◽  
Vol 81 (4) ◽  
pp. 767-776 ◽  
Author(s):  
Cinzia Chiappe ◽  
Marco Malvaldi ◽  
Christian Silvio Pomelli
Keyword(s):  
Ionic Liquids ◽  
Kinetic Data ◽  
Classification Criteria ◽  
Experimental Measurements ◽  
Molecular Liquids ◽  
The Poor ◽  
Poor Understanding ◽  
The Many ◽  
Molecular Solvents

The role of ionic liquids (ILs) as solvents in chemistry is limited by the poor understanding of the solvation phenomenon in these media. The usual classification criteria used for molecular solvents through various experimental measurements fail to insert ILs into a univocal classification for ILs. Here, we first discuss the unsuitability of the usual interpretative scheme for molecular liquids and elucidate schematically the mechanism of solvation in ILs, pointing out the peculiarities that differentiate them with respect to molecular liquids. Second, we focus on reactivity and reaction kinetics in ILs, underlining the many problems that the complexity of these media reflects on the interpretation of kinetic data and some possible approaches to understand qualitatively the (often not trivial) kinetic problems for reactions performed in ILs.

Elevated antigen-specific Th2 type response is associated with the poor prognosis of hand, foot and mouth disease

2013 ◽  
Vol 177 (1) ◽  
pp. 62-65 ◽  
Author(s):  
Ruicheng Wei ◽  
Lijuan Xu ◽  
Na Zhang ◽  
Kai Zhu ◽  
Juhao Yang ◽  
...  
Keyword(s):  
Poor Prognosis ◽  
Foot And Mouth Disease ◽  
Mouth Disease ◽  
The Poor ◽  
Foot And Mouth ◽  
Type Response

scholarly journals Evaluation of serum ATX and LPA as potential diagnostic biomarkers in patients with pancreatic cancer

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Jiang Chen ◽  
Hongyu Li ◽  
Wenda Xu ◽  
Xiaozhong Guo
Keyword(s):  
Pancreatic Cancer ◽  
Poor Prognosis ◽  
Prognostic Value ◽  
Potential Role ◽  
Early Stage ◽  
Serum Levels ◽  
Diagnostic Efficacy ◽  
Diagnostic Biomarkers ◽  
The Poor

Abstract Background Pancreatic cancer (PC) is a devastating disease that has a poor prognosis and a total 5-year survival rate of around 5%. The poor prognosis of PC is due in part to a lack of suitable biomarkers that can allow early diagnosis. The lysophospholipase autotaxin (ATX) and its product lysophosphatidic acid (LPA) play an essential role in disease progression in PC patients and are associated with increased morbidity in several types of cancer. In this study, we evaluated both the potential role of serum LPA and ATX as diagnostic markers in PC and their prognostic value for PC either alone or in combination with CA19-9. Methods ATX, LPA and CA19-9 levels were evaluated using ELISA of serum obtained from PC patients (n = 114) healthy volunteers (HVs: n = 120) and patients with benign pancreatic diseases (BPDs: n = 94). Results Serum levels of ATX, LPA and CA19-9 in PC patients were substantially higher than that for BPD patients or HVs (p < 0.001). The sensitivity of LPA in early phase PC was 91.74% and the specificity of ATX was 80%. The levels of ATX, LPA and CA19-9 were all substantially higher for early stage PC patients compared to levels in serum from BPD patients and HVs. The diagnostic efficacy of CA19-9 for PC was significantly enhanced by the addition of ATX and LPA (p = 0.0012). Conclusion Measurement of LPA and ATX levels together with CA19-9 levels can be used for early detection of PC and diagnosis of PC in general.

Clinical Translation, Ethics, and Policy

2021 ◽  
Author(s):  
Jonathan Kimmelman
Keyword(s):  
Clinical Practice ◽  
Conceptual Framework ◽  
Linear Process ◽  
Clinical Translation ◽  
Ethical Aspects ◽  
New Treatments ◽  
Relationship Of ◽  
The Relationship ◽  
Translation Ethics

Many representations of clinical translation present it as a linear process by which bench discoveries are advanced into clinical practice. The present chapter offers a conceptual framework that highlights distinctive ethical aspects of clinical translation. The framework understands clinical translation as a process not so much as developing new treatments but rather generating evidence on how to use (and how not to use) new as well as old interventions. It also emphasizes attending to the relationship of different studies with each other and the importance of coordinating different studies with each other. The model presented here foregrounds the role of coordination in realizing societal objectives of clinical translation at minimal burden and cost for patients and study volunteers.

Dedifferentiated chondrosarcoma: A report of the clinicopathologic features and outcomes of 16 cases treated at a single institution.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e23500-e23500
Author(s):  
Charles Gusho ◽  
Steven Gitelis ◽  
Alan T. Blank
Keyword(s):  
Poor Prognosis ◽  
Nonoperative Management ◽  
Clinicopathological Features ◽  
Clinicopathologic Features ◽  
Single Institution ◽  
Dedifferentiated Chondrosarcoma ◽  
Wide Margin ◽  
The Poor ◽  
Kaplan Meier ◽  
Period Data

e23500 Background: Dedifferentiated chondrosarcoma (DCS) is a rare and aggressive malignancy with a poor prognosis. The purpose of this investigation was to assess the clinicopathological features and outcomes of DCS patients treated at a single institution. Methods: This study was a retrospective review over a consecutive twenty-year period. Data including treatment details and outcomes were recorded. Results: A total of 16 cases from 2000 to 2018 were reviewed. The median age was 62 years (IQR, 52-69 years) and the majority of DCS arose in the femur (50%, n = 8) and pelvis (25%, n = 4). Fourteen (88%) cases received limb salvage/wide margin resection (n = 13) or intralesional surgery (n = 1). For all DCS, the median estimated overall survival (OS) was 46 months (95% CI, 1-90 months) with both a five and ten-year survival probability of 32%. On Kaplan-Meier analysis there was no difference between operative versus nonoperative management (p = 0.747), surgery alone versus surgery/chemotherapy (p = 0.265), nor surgery alone versus surgery/chemotherapy/radiation (p = 0.698). Conclusions: Our findings confirm the poor prognosis of DCS patients, though with a five-year estimate of 32%, higher than previous literature. Together with existing literature, our data may enable future strategic recommendation of DCS patients.

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