scholarly journals Place in therapy of innovative drugs in multiple myeloma in 2021 and 2023 according to an expert panel Delphi consensus

Author(s):  
Mario Boccadoro ◽  
Patrizia Berto ◽  
Sara Bringhen ◽  
Elena Zamagni ◽  
Patrizia Tosi ◽  
...  

Introduction: The objective of this study was to understand the potential use of single agents and drug combinations in multiple myeloma (MM) across treatment lines in the years 2021 and 2023. Methods: The method used was Delphi Panel Method survey, administered to European Myeloma Network (EMN) Italy Working Group centres. Future treatments were identified assessing all available web-based information sources, including therapies (single drugs or combinations) with strong evidence of efficacy, likely to be on the Italian market in 2021 and 2023. Participants were asked to report on the likelihood of prescription for MM therapies, across treatment lines. Results: Across the 15 centres taking part in the survey, about 890 patients per year are forecasted to receive a new diagnosis of MM. In 2021, the Panel forecasted 66% of 1L-TE (transplant eligible) patients will be treated with bortezomib-thalidomide-dexamethasone (VTD) and 32% of patients with daratumumab-bortezomib-thalidomide-dexamethasone (DVTd), with a substantial decrease of VTD (15%) and a marked increase of DVTd (81%) forecasted for 2023. The 2L and 3L R(lenalidomide)-based combination treatments are expected to drop and will likely be substituted by a steep increase in P(pomalidomide)-based regimes (from 7% to 23%). On the contrary, in 3L treatment, all combination therapies (with the exception of IsaPd – isatuximab-pomalidomide-dexamethasone) are expected to lose market share in favour of the most recent new therapies. Conclusions: Expert Panel agrees that many different new drugs and combinations will be used in MM, with different mechanisms of action, both at diagnosis and in subsequent phases of the disease, with a corresponding decline of the drugs currently used.

Toxins ◽  
2021 ◽  
Vol 13 (4) ◽  
pp. 236
Author(s):  
Alessio Baricich ◽  
Theodore Wein ◽  
Nicoletta Cinone ◽  
Michele Bertoni ◽  
Alessandro Picelli ◽  
...  

There is extensive literature supporting the efficacy of botulinum toxin (BoNT-A) for the treatment of post-stroke spasticity, however, there remain gaps in the routine management of patients with post-stroke spasticity. A panel of 21 Italian experts was selected to participate in this web-based survey Delphi process to provide guidance that can support clinicians in the decision-making process. There was a broad consensus among physicians that BoNT-A intervention should be administered as soon as the spasticity interferes with the patients’ clinical condition. Patients monitoring is needed over time, a follow-up of 4–6 weeks is considered necessary. Furthermore, physicians agreed that treatment should be offered irrespective of the duration of the spasticity. The Delphi consensus also stressed the importance of patient-centered goals in order to satisfy the clinical needs of the patient regardless of time of onset or duration of spasticity. The findings arising from this Delphi process provide insights into the unmet needs in managing post-stroke spasticity from the clinician’s perspective and provides guidance for physicians for the utilization of BoNT-A for the treatment of post-stroke spasticity in daily practice.


Author(s):  
Antonia S. Stang ◽  
Melanie Trudeau ◽  
Otto G. Vanderkooi ◽  
Bonita E. Lee ◽  
Linda Chui ◽  
...  

Background. We sought to develop diagnostic test guidance definitions for pediatric enteric infections to facilitate the interpretation of positive test results in the era of multianalyte molecular diagnostic test platforms.Methods. We employed a systematic, two-phase, modified Delphi consensus process consisting of three web-based surveys and an expert panel face-to-face meeting. In phase 1, we surveyed an advisory panel of North American experts to select pathogens requiring diagnostic test guidance definition development. In phase 2, we convened a 14-member expert panel to develop, refine, and select the final definitions through two web-based questionnaires interspersed with a face-to-face meeting. Both questionnaires asked panelists to rate the degree to which they agreed that if the definition is met the pathogen is likely to be causative of clinical illness.Results. The advisory panel survey identified 19 pathogens requiring definitions. In the expert panel premeeting survey, 13 of the 19 definitions evaluated were rated as being highly likely (“agree” or “strongly agree”) to be responsible for acute gastroenteritis symptoms by ≥67% of respondent panel members. The definitions for the remaining six pathogens (Aeromonas, Clostridium difficile, Edwardsiella,nonenteric adenovirus, astrovirus, andEntamoeba histolytica) were indeterminate. After the expert panel meeting, only two of the modified definitions,C. difficileandE. histolytica/dispar, failed to achieve thea priorispecified threshold of ≥67% agreement.Conclusions.We developed diagnostic test guidance definitions to assist healthcare providers for 17 enteric pathogens. We identified two pathogens that require further research and definition development.


Author(s):  
Victor L Perez ◽  
Juan Carlos Abad ◽  
José Alvaro P Gomes

ABSTRACT Purpose To establish consensus on definitions and concepts of keratoconus and ectatic corneal diseases by using Delphi approach. Materials and methods Seventeen Latin American experts on keratoconus and ectatic corneal diseases participated in a 3-round Delphi panel approach. A web-based 12 question survey was answered by panelists in two rounds, followed by life discussion meeting. A two-third majority strategy was defined as a consensus. Results Corneal ectatic diseases were defined as progressive thinning conditions without acute stromal inflammation, and were classified as keratoconus, pellucid marginal degeneration (PMD), keratoglobus, and ectasia after trauma or surgery. The pattern of thinning was considered the main aspect for the classification of ectatic diseases, but criteria for staging the disease needs to be improved. Corneal tomography with 3D reconstruction, distinguished from Placido-disk based front surface topography was elected as the diagnostic procedure currently capable of detecting the earliest clinical ectatic changes. Biomechanical alterations were considered to be the first event for ectasia development. Eye rubbing was directly related to the development and progression of ectasia. Inflammation was considered as a possible event on the physiopathology. Keratoconus was considered as a bilateral disease, with often asymmetric presentation, while ectasia may occur unilaterally if there is significant corneal insult, leading to biomechanical failure. No consensus was achieved for the definitions on forme fruste keratoconus (FFKC). Conclusion Consensus was achieved on definitions of corneal ectatic diseases. Disease staging, including criteria for FFKC or subclinical ectasia remains controversial. A larger consensus including experts from different groups around the world is needed. How to cite this article Ambrósio RJr, Belin MW, Perez VL, Abad JC, Gomes JAP. Definitions and Concepts on Keratoconus and Ectatic Corneal Diseases: Panamerican Delphi Consensus — A Pilot for the Global Consensus on Ectasias. Int J Kerat Ect Cor Dis 2014;3(3):99-106.


2020 ◽  
Vol 51 (3) ◽  
pp. 120-124
Author(s):  
Dominik Dytfeld

AbstractIn spite of the introduction of several new drugs in the last 10 years, multiple myeloma (MM) remains incurable. Thus, an adoptive cellular therapy using chimeric antigen receptor T (CART), a strategy to increase the frequency of tumor-directed and functionally active T cells targeting antigens present on the cancer cell, might change the treatment in MM as it did in lymphoma and ALL. There are several targets for CART therapy in MM on different levels of development, which are discussed in the manuscript. B-cell maturation antigen (BCMA) being tested in the studies of phase 1–2 is the most promising, but so far CART has not been approved in the cure of MM and remains an experimental approach. The hematological society is facing a new technology which with its potential ability to cure MM, in spite of its complexity, cost, and toxicity, will definitely and soon change the landscape of myeloma in Europe and world-wide.


Cancers ◽  
2021 ◽  
Vol 13 (14) ◽  
pp. 3523
Author(s):  
Wancheng Guo ◽  
Haiqin Wang ◽  
Peng Chen ◽  
Xiaokai Shen ◽  
Boxin Zhang ◽  
...  

Multiple myeloma (MM) is a B-cell tumor of the blood system with high incidence and poor prognosis. With a further understanding of the pathogenesis of MM and the bone marrow microenvironment, a variety of adjuvant cell therapies and new drugs have been developed. However, the drug resistance and high relapse rate of MM have not been fundamentally resolved. Studies have shown that, in patients with MM, there is a type of poorly differentiated progenitor cell (MM stem cell-like cells, MMSCs). Although there is no recognized standard for identification and classification, it is confirmed that they are closely related to the drug resistance and relapse of MM. This article therefore systematically summarizes the latest developments in MMSCs with possible markers of MMSCs, introduces the mechanism of how MMSCs work in MM resistance and recurrence, and discusses the active pathways that related to stemness of MM.


2014 ◽  
Vol 26 (2) ◽  
pp. 128-133 ◽  
Author(s):  
Giampiero Girolomoni ◽  
Gianfranco Altomare ◽  
Fabio Ayala ◽  
Enzo Berardesca ◽  
Piergiacomo Calzavara Pinton ◽  
...  

2008 ◽  
Vol 9 (12) ◽  
pp. 1157-1165 ◽  
Author(s):  
Enrique M Ocio ◽  
María-Victoria Mateos ◽  
Patricia Maiso ◽  
Atanasio Pandiella ◽  
Jesús F San-Miguel

2018 ◽  
Vol Volume 12 ◽  
pp. 2387-2396 ◽  
Author(s):  
Thomas Wilke ◽  
Sabrina Mueller ◽  
Sabine Bauer ◽  
Silvia Pitura ◽  
Leona Probst ◽  
...  

10.2196/14744 ◽  
2020 ◽  
Vol 9 (1) ◽  
pp. e14744
Author(s):  
Vishal Vennu ◽  
Saurabh Dahiya

Background Although several studies have been conducted and several articles have been published on India's new clinical trial regulations, very few have examined the views of investigators and ethics board members regarding modifications to the previous regulations. Overall, they have neglected to find out the opinions of other relevant professionals, such as research assistants, coordinators, associates, and managers. To our knowledge, no study has yet investigated the awareness and opinions of Indian research professionals on the new 2019 regulations. Objective This study aims to describe the awareness and opinions of Indian research professionals on the new drug and clinical trial regulations. Methods In this cross-sectional, Web-based study, we will conduct an open survey for various Indian research professionals. These professionals will be selected randomly using multiple sources. The survey questionnaires, which have already been validated, were developed using the form function in Google docs. A Web link was generated for participants to take the survey. Descriptive statistics will be shown as means and standard deviations for constant variables, whereas certain variables will instead be shown as numbers and percentages. Results The survey was opened in July 2019. Enrollment has already started and will be completed in three months. The results calculations are expected to begin in October 2019. Conclusions The results of the survey are expected to represent the views of research professionals on the new regulations that will support the development of clinical research and the pharmaceutical industry in India. These regulations are expected to help advance clinical trials, help with the approval of new drugs, and enhance ethical norms in the country. International Registered Report Identifier (IRRID) PRR1-10.2196/14744


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