Can pediatric and adolescent patients with recurrent tumors benefit from a precision medicine program? The European MAPPYACTS experience.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 10018-10018 ◽  
Author(s):  
Pablo Berlanga ◽  
Gaëlle Pierron ◽  
Ludovic Lacroix ◽  
Tiphaine Adam de Beaumais ◽  
Virginie Bernard ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Precision Medicine ◽  
Tumor Board ◽  
Molecular Profile ◽  
Proof Of Concept ◽  
Tumor Biopsy ◽  
Rapid Disease Progression ◽  
Targeted Agent

10018 Background: The international prospective precision medicine trial MAPPYACTS (NCT02613962) aims to define the molecular profile in recurrent/refractory malignancies in order to suggest the most adapted salvage treatment. Methods: Patients < 18 years-old at the time of initial diagnosis underwent on-purpose tumor biopsy/surgery of their recurrent/refractory malignancy for molecular characterization by WES and whole RNA sequencing. Results were reviewed in a dedicated weekly molecular tumor board (MTB), followed by discussion with the treating physician in a clinical MTB (CMTB). Whenever possible, patients were enrolled subsequently in clinical trials based on CMTB recommendations. Results: From February 2016 to October 2018, 500 patients have been included in 17 centers in France, Italy and Ireland. Median age at inclusion was 13 years. 38% had sarcomas, 28% brain tumors, 22% other solid tumors, 12% hematological malignancies with a median of one prior relapse/progression. Eleven patients did not undergo intervention procedure (10 screening failures, 1 consent withdrawal). Molecular profiling was performed on samples in 433 patients and was contributive for 390 patients. For 271/390 patients (70%), there was at least one genetic alteration that could represent a potential therapeutic target and 8% had alterations considered as “ready for use” for treatment at relapse. Among them, 19 (7%) died before the CMTB. With follow-up censored in January 2019, 72 patients (27%) were treated with at least one matched targeted agent, 57 of them in a clinical trial, mostly the proof-of concept AcSé-ESMART trial (NCT02813135). Of the 166 patients (61%) that did not receive the matched treatment recommended by CMTB (80 still on follow-up), main reasons were: response/stable disease under previous therapy (50), another treatment (48), rapid disease progression/death (30) and legal representative refusal (12). Conclusions: MAPPYACTS profiling allowed tailored targeted treatment, mainly through early clinical trials, in patients with recurrent pediatric cancer. Since most detected molecular findings are within the investigational or hypothetical evidence level, therapeutic proof-of-concept trials that are exploring targeted therapies in molecularly enriched patient populations are crucial to improve knowledge and potentially outcome. Clinical trial information: NCT02613962.

Clinical utility of molecular testing to select therapy in relapsed/refractory non-Hodgkin lymphoma: Mayo Clinic Center for Individualized Medicine experience.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 11571-11571
Author(s):  
Nabila Nora Bennani ◽  
Stephen Maxted Ansell ◽  
Thomas E. Witzig ◽  
Andew L. Feldman ◽  
Tammy M McAllister ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Targeted Therapy ◽  
Precision Medicine ◽  
Mayo Clinic ◽  
Individualized Medicine ◽  
Tumor Board ◽  
Current Therapy ◽  
Molecular Profile ◽  
Molecular Testing ◽  
Molecular Alterations

11571 Background: Relapsed/refractory (R/R) non-Hodgkin lymphomas (NHL) have a poor prognosis with limited treatment options. Our expanding knowledge of molecular alterations seen in R/R NHL allows identification of patients that potentially may benefit from a precision medicine approach. However, experience in routine clinical implementation of precision medicine has been limited. Here, we summarize our clinical experience in molecular characterization of RR NHL targeted therapy (TT) using next-generation sequencing (NGS), and selection of targeted therapy (TT) based on molecular profile. Methods: We conducted a prospective study in RR NHL through the Center for Individualized Medicine at Mayo Clinic. Consenting patients underwent NGS using FoundationOne Heme panel from biopsies done at time of relapse. Results of NGS were discussed at the Genomic Tumor Board and recommendations for TT were given based on matching specific molecular alteration(s) with potential agent(s) predicted to be active based on NGS. The agents could include FDA-approved, off-label use and clinical trial therapies. Results: 28 cases were enrolled: 18 aggressive NHL, 10 follicular lymphoma (FL). Molecular alterations were present in all cases. In aggressive B-cell NHL, CDKN2A/B gene cluster alterations were seen in 73% (8/11), while seen in only 1/7 T-cell lymphomas (TCL), and 1/10 FL. TP53 deletions were second most common genomic alterations in DLBCL (57%) and seen in 40% FL. JAK-STAT and ERBB pathways were altered in TCL (2/7 each). IGH-BCl-2 gene rearrangement were common in FL (70%), followed by MLL gene alterations (50%). Targetable mutations were present in 86% (24/28) of cases. A TT was recommended in all 24 cases, but received by 2 patients only. Remaining patients did not due to benefit from current therapy (10/24), ineligibility or lack of clinical trial (7/24) or interim clinical deterioration (5/24). Conclusions: Targetable mutations were identified in most cases of RR NHL with TT recommended for all cases. However, access to TT limits potential clinical benefit of molecular-based matching strategy. More studies are needed to assess impact on clinical outcomes.

scholarly journals SO093LONGITUDINAL DATA ON TREATMENT DURATION AND COMPLIANCE FROM AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE CLINICAL TRIALS WITH TOLVAPTAN

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Xiaolei Zhou ◽  
Diana Garbinsky ◽  
John Ouyang ◽  
Eric Davenport ◽  
Indra Agarwal ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Treatment Duration ◽  
Treatment Time ◽  
Treatment Compliance ◽  
Kidney Volume ◽  
Total Kidney Volume ◽  
Treatment Gaps ◽  
Clinical Trial Program

Abstract Background and Aims : Observation of impactful clinical outcomes in a clinical trial setting for ADPKD is challenging due to the life-long progressive nature of ADPKD and longer-term associated outcomes of interest in this population (e.g., renal function decline, cardiovascular events, and mortality). Since 2004, the tolvaptan (TOL) clinical trial program enrolled subjects in multiple clinical studies with the opportunity to enroll in subsequent clinical trials for treatment and outcomes evaluation. Method : Data from 6 ADPKD studies (protocols 156-04-250, 156-04-251, 156-06-260, 156-09-284, 156-09-290, 156-08-271) were pooled and evaluated over time for overall treatment duration, treatment time, and treatment gaps. Treatment duration for the individual clinical trials ranged from 1 week to up to 3 years. Results : Overall, 1,437 subjects received TOL in these ADPKD clinical trials. For these subjects, the mean overall treatment duration was 4.1 years (3.8 years on treatment) with a maximum of 9.7 years (9.0 years on treatment). In this cohort, 513 subjects (35.7%) received TOL treatment for more than 5 years. Mean treatment compliance was 94.1%. Overall, 723 subjects (50.3%) received TOL treatment in ≥2 trials, with a median treatment gap duration between trials of 0.1 years (maximum, 5.6 years). At least 7 years of follow-up data are available for estimated glomerular filtration rate in 241 subjects (mean at baseline, 78.6 mL/min/1.73m2) and for total kidney volume in 130 subjects (mean at baseline, 1,816.9 mL). Conclusion : This analysis provides longitudinal follow-up over an extended timeframe in a large number of subjects treated with TOL, with the greatest number of subjects being enrolled in clinical trials enriched for rapidly progressing ADPKD. Treatment compliance over years was reasonably good despite treatment gaps.

Dedicated clinical trial tissue tracking database to improve turn-around time at high-volume center.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 1543-1543
Author(s):  
Peter Blankenship ◽  
David DeLaRosa ◽  
Marc Burris ◽  
Steven Cusson ◽  
Kayla Hendricks ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Tissue Sample ◽  
High Volume ◽  
Trial Participation ◽  
Fresh Tissue ◽  
Oncology Clinical Trials ◽  
The Status ◽  
Unique Source

1543 Background: Tissue requirements in oncology clinical trials are increasingly complex due to prescreening protocols for patient selection and serial biopsies to understand molecular-level treatment effects. Novel solutions for tissue processing are necessary for timely tissue procurement. Based on these needs, we developed a Tissue Tracker (TT), a comprehensive database for study-related tissue tasks at our high-volume clinical trial center. Methods: In this Microsoft Access database, patients are assigned an ID within the TT that is associated with their name, medical record number, and study that follows their request to external users: pathology departments, clinical trial coordinators and data team members. To complete tasks in the TT, relevant information is required to update the status. Due to the high number of archival tissue requests from unique pathology labs, the TT has a “Follow-Up Dashboard” that organizes information needed to conduct follow-up on all archival samples with the status “Requested”. This results in an autogenerated email and pdf report sent to necessary teams. The TT also includes a kit inventory system and a real-time read only version formatted for interdepartmental communication, metric reporting, and other data-driven efforts. The primary outcome in this study was to evaluate our average turnaround time (ATAT: average time from request to shipment) for archival and fresh tissue samples before and after TT development. Results: Before implementing the TT, between March 2016 and March 2018, we processed 2676 archival requests from 235 unique source labs resulting in 2040 shipments with an ATAT of 19.29 days. We also processed 1099 fresh biopsies resulting in 944 shipments with an ATAT of 7.72 days. After TT implementation, between April 2018 and April 2020, we processed 2664 archival requests from 204 unique source labs resulting in 2506 shipments (+28.0%) with an ATAT of 14.78 days (-23.4%). During that same period, we processed 1795 fresh biopsies (+63.3%) resulting in 2006 shipments (+112.5%) with an ATAT of 6.85 days (-11.3%). Conclusions: Oncology clinical trials continue to evolve toward more extensive tissue requirements for prescreening and scientific exploration of on-treatment molecular profiling. Timely results are required to optimize patient trial participation. During the intervention period, our tissue sample volume and shipments increased, but the development and implementation of an automated tracking system allowed improvement in ATAT of both archival and fresh tissue. This automation not only improves end-user expectations and experiences for patients and trial sponsors but this allows our team to adapt to the increasing interest in tissue exploration.

scholarly journals Hemophilia gene therapy: ushering in a new treatment paradigm?

Hematology ◽  
2021 ◽  
Vol 2021 (1) ◽  
pp. 226-233
Author(s):  
Lindsey A. George
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Clinical Trials ◽  
Hemophilia A ◽  
Full Potential ◽  
Proof Of Concept ◽  
Aav Vector ◽  
Treatment Paradigm ◽  
Clinical Trial Enrollment ◽  
New Treatment

Abstract After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within approximately 1 year. While supported optimism suggests that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized, a number of outstanding questions have emerged from clinical trial that are in need of answers to harness the full potential of gene therapy for hemophilia patients. This article reviews the use of AAV vector gene addition approaches for hemophilia A and B, focusing specifically on information to review in the process of obtaining informed consent for hemophilia patients prior to clinical trial enrollment or administering a licensed AAV vector.

scholarly journals Results availability and timeliness of registered COVID-19 clinical trials: interim cross-sectional results from the DIRECCT study

BMJ Open ◽  
2021 ◽  
Vol 11 (11) ◽  
pp. e053096
Author(s):  
Maia Salholz-Hillel ◽  
Peter Grabitz ◽  
Molly Pugh-Jones ◽  
Daniel Strech ◽  
Nicholas J DeVito
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Journal Article ◽  
Cross Sectional Study ◽  
Sensitivity Analyses ◽  
Cross Sectional ◽  
Registration Data ◽  
Clinical Trial Registries ◽  
Treatment And Prevention

ObjectiveTo examine how and when the results of COVID-19 clinical trials are disseminated.DesignCross-sectional study.SettingThe COVID-19 clinical trial landscape.Participants285 registered interventional clinical trials for the treatment and prevention of COVID-19 completed by 30 June 2020.Main outcome measuresOverall reporting and reporting by dissemination route (ie, by journal article, preprint or results on a registry); time to reporting by dissemination route.ResultsFollowing automated and manual searches of the COVID-19 literature, we located 41 trials (14%) with results spread across 47 individual results publications published by 15 August 2020. The most common dissemination route was preprints (n=25) followed by journal articles (n=18), and results on a registry (n=2). Of these, four trials were available as both a preprint and journal publication. The cumulative incidence of any reporting surpassed 20% at 119 days from completion. Sensitivity analyses using alternate dates and definitions of results did not appreciably change the reporting percentage. Expanding minimum follow-up time to 3 months increased the overall reporting percentage to 19%.ConclusionCOVID-19 trials completed during the first 6 months of the pandemic did not consistently yield rapid results in the literature or on clinical trial registries. Our findings suggest that the COVID-19 response may be seeing quicker results disclosure compared with non-emergency conditions. Issues with the reliability and timeliness of trial registration data may impact our estimates. Ensuring registry data are accurate should be a priority for the research community during a pandemic. Data collection is underway for the next phase of the DIssemination of REgistered COVID-19 Clinical Trials study expanding both our trial population and follow-up time.

scholarly journals Patients with cancer in the COVID-19 era: the clinical trial issue

2020 ◽  
Vol 106 (4) ◽  
pp. 271-272 ◽  
Author(s):  
Marcello Scarcia ◽  
Giuseppe Mario Ludovico ◽  
Angela Fortunato ◽  
Alba Fiorentino
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Physical Examination ◽  
Regulatory Agencies ◽  
Study Medication ◽  
Therapeutic Modalities ◽  
Patients With Cancer ◽  
Safety Guidelines ◽  
Trial Discontinuation

Coronavirus disease 2019 (COVID-19) hospital reorganization may result in reduced ability for the hospital to fully use its armamentarium for battling cancer. Thus different therapeutic modalities have been recommended. During the pandemic, despite regulatory agencies’ recommendations, several considerations and doubts remain for oncologic clinical trials. Considering patients who had been enrolled before the pandemic, and who plan to take the study medication, the situation becomes complicated. These patients should undergo monitoring visits, blood sampling, questionnaire, physical examination, and drug and radiation administration. To avoid deviations from the protocol and trial discontinuation, follow-up should be performed regularly, in concordance with safety guidelines. Here we report several considerations.

scholarly journals Parental perspectives long term after neonatal clinical trial participation: a survey

Trials ◽  
2020 ◽  
Vol 21 (1) ◽  
Author(s):  
Thomas Salaets ◽  
Emilie Lavrysen ◽  
Anne Smits ◽  
Sophie Vanhaesebrouck ◽  
Maissa Rayyan ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Trial Participation ◽  
Drug Trials ◽  
Clinical Trial Participation ◽  
Parental Perspectives ◽  
Positive Experiences ◽  
Almost All

Abstract Background Although recruiting newborns is ethically challenging, clinical trials remain essential to improve neonatal care. There is a lack of empirical data on the parental perspectives following participation of their neonate in a clinical trial, especially at long term. The objective of this study is to assess experiences and emotions of parents, long term after trial participation in an interventional drug trial. Methods Parents of former participants of five neonatal interventional drug trials were surveyed at long term (3–13 years ago) after participation. The survey assessed parental contentment with trial participation, perceived influence of the trial on care and health, emotional consequences of participation, and awareness of typical clinical trial characteristics on 6-point Likert scales. Results Complete responses were received from 123 parents (52% of involved families). Twenty percent of parents did not remember participation. Those who remembered participation reported high contentment with overall trial participation (median 5.00), but not with follow-up (median 3.00). Most parents did not perceive any influence of the trial on care (median 2.00) and health (median 2.43). Almost all parents reported satisfaction and pride (median 4.40), while a minority of parents reported anxiety and stress (median 1.44) or guilt (median 1.33) related to trial participation. A relevant minority was unaware of typical trial characteristics (median 4.20; 27% being unaware). Conclusions Overall, parents reported positive experiences and little emotional distress long term after participation. Future efforts to improve the practice of neonatal clinical trials should focus on ensuring effective communication about the concept and characteristics of a clinical trial during consent discussions and on the follow-up after the trial.

scholarly journals The evolution of clinical trials: Can we address the challenges of the future?

2018 ◽  
Vol 15 (1_suppl) ◽  
pp. 27-32 ◽  
Author(s):  
Hans-Georg Eichler ◽  
Fergus Sweeney
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Precision Medicine ◽  
External Validity ◽  
Pragmatic Trials ◽  
The Future ◽  
Meaningful Data

The authors describe key challenges facing the clinical trials community and propose solutions to these issues, including the role the Clinical Trials Transformation Initiative can play in addressing these issues. Specifically, the authors reflect on clinical trial globalization and the harmonization of frameworks and requirements across regions; the challenges associated with balancing the desire for external validity, pragmatic trials, and precision medicine; clinical trial transparency; and operational complexity and the expense of clinical trials. By addressing these challenges, future clinical trials will be more feasible, relevant, and credible, and support both the continuing altruistic contributions of patients and the collection of more meaningful data.

scholarly journals Individualized Molecular Profiling for Allocation to Clinical Trials Singapore Study—An Asian Tertiary Cancer Center Experience

2021 ◽  
pp. 859-875
Author(s):  
Amanda O. L. Seet ◽  
Aaron C. Tan ◽  
Tira J. Tan ◽  
Matthew C. H. Ng ◽  
David W. M. Tai ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Tumor Tissue ◽  
Molecular Profiling ◽  
Tumor Board ◽  
Cancer Center ◽  
Precision Oncology ◽  
Molecular Tumor Board ◽  
Tumor Types ◽  
Tertiary Cancer Center

PURPOSE Precision oncology has transformed the management of advanced cancers through implementation of advanced molecular profiling technologies to identify increasingly defined subsets of patients and match them to appropriate therapy. We report outcomes of a prospective molecular profiling study in a high-volume Asian tertiary cancer center. PATIENTS AND METHODS Patients with advanced cancer were enrolled onto a prospective protocol for genomic profiling, the Individualized Molecular Profiling for Allocation to Clinical Trials Singapore study, at the National Cancer Center Singapore. Primary objective was to identify molecular biomarkers in patient's tumors for allocation to clinical trials. The study commenced in February 2012 and is ongoing, with the results of all patients who underwent multiplex next-generation sequencing (NGS) testing until December 2018 presented here. The results were discussed at a molecular tumor board where recommendations for allocation to biomarker-directed trials or targeted therapies were made. RESULTS One thousand fifteen patients were enrolled with a median age of 58 years (range 20-83 years). Most common tumor types were lung adenocarcinoma (26%), colorectal cancer (15%), and breast cancer (12%). A total of 1,064 NGS assays were performed, on fresh tumor tissue for 369 (35%) and archival tumor tissue for 687 (65%) assays. TP53 (39%) alterations were most common, followed by EGFR (21%), KRAS (14%), and PIK3CA (10%). Of 405 NGS assays with potentially actionable alterations, 111 (27%) were allocated to a clinical trial after molecular tumor board and 20 (4.9%) were enrolled on a molecularly matched clinical trial. Gene fusions were detected in 23 of 311 (7%) patients tested, including rare fusions in new tumor types and known fusions in rare tumors. CONCLUSION Individualized Molecular Profiling for Allocation to Clinical Trials Singapore demonstrates the feasibility of a prospective broad molecular profiling program in an Asian tertiary cancer center, with the ability to develop and adapt to a dynamic landscape of precision oncology.

scholarly journals Epidemiology of Adults AML in Nord-Pas De Calais and Picardy

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. 2281-2281
Author(s):  
Christophe Roumier ◽  
Céline Rodriguez ◽  
Cécile Frimat ◽  
Céline Berthon ◽  
Carole Delattre ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Molecular Biology ◽  
Median Survival ◽  
Conflicts Of Interest ◽  
Real Life ◽  
Npm1 Mutation ◽  
Demethylating Agent ◽  
Molecular Abnormalities

Abstract Introduction AML is a heterogeneous group of neoplasm according to leukemogenic mechanisms and has variable response to treatment. In addition to age and WBC count at diagnosis, cytogenetic and molecular abnormalities are key factors to assess prognosis. In this work, we evaluated the number of adult AML cases diagnosed between 2009 and 2012 in the French Nord Pas de Calais and Picardie regions (North of France). The aim of the study was the collection of epidemiology data, treatments proposed and their results in real life. Matérials and Methods The Nord Pas de Calais and Picardie observatory contains data of all adult patients (>18 years) with AML (>20% marrow blasts) diagnosed in the oncology and/or hematology departments of the following participating Hospitals: Amiens (A), Arras (Ar), Boulogne (B), Dunkerque (D), Lens (L), Lille (CHRU et CH St Vincent (Vi)), Roubaix (R) et Valenciennes (V), whose overall catchment population is close to 6 million inhabitants , allowing a population based registry. A disadvantage of registry data is that investigators cannot control data collection and quality. In our observatory, data were obtained from the declaration of all the participants centralized with a web access reporting data base. The declaration of patients in the database was made both by clinicians, cytogeneticists and molecular biologists. The following data were entered:, disease course, WBC counts, bone marrow blast %, cytogenetics, immunophenotype, molecular biology, treatment received, outcome. To optimize data quality, each kind of data was entered respectively by clinicians, cytogeneticists and molecular biologists. Data were cross validated and the follow up data were updated every years during follow-up of the patients. Data was collected as of October 2013 Results Between January 2009 and December 2012, 899 patients were enrolled in the observatory, respectively 185 (A), 33 (Ar), 43 (B), 66 (D), 85 (L), 197 (CHRU), 57 (Vi), 98 (R) and 135 (V). Median age was 67 years (range 17 to 97), 54% of patients were older than 65 years, M/F was 1.12 (425 female (47%) et 474 male (53%)). Cytogenetic studies were performed in 87% of the patients, immunophenotype in 57%, molecular biology screening for FLT3 and NPM1 mutation in 61%. Intent of treatment was conventional chemotherapy (CTi) 50%, demethylating agent (DMT) 18% and best supportive care (BSC) 32% + Low dose cytarabine (LDAC). The overall median survival was 269 days (624d < 65 y and 115d for > 65 y) (fig1). 15% of the patients (132pts) were included in a clinical trial (90 % <65 y and 10% >65 y). Median overall survival of the patients included in a clinical trial and aged <65y was 1121d vs 563d for patients not included in a clinical trial and aged <65y (p:ns). Of the 482 patients aged >65 y, MRC cytogenetic classification was unfavourable (139 pts; 29%), intermediate (209 pts; 43%), favourable (18 pts; 4%) and not done 116 pts (24%). Overall, in pts aged > 65, treatment was CTI 93 pts (19%), DMT 130 pts (27%), BSC 210 pts (44%), LDAC) 38 pts (8%), others 11 pts (2%). Among pts with unfavourable karyotype treatment was: Cti (14%), DMT (45%), LDAC (2%), BSC (38%) and others (1%). overall median survival in this unfavourable group was 222d with DMT vs 104d with LDAC vs 227d with CTI. In intermediate cytogenetics cases, treatment was Cti (24%), DMT (19%), LDAC (14%), BSC (42%) and other (1%), and median OS was 270d with DMT vs 189d with ARAC-LD vs 378d with Cti. Demethylating agent treatment results were different between MRC groups. In the intermediate MRC group of pts >65y, OS was better with CTI vs DMT (p:0.03), whereas in the unfavorable pts group aged >65y/o, DMT treatment seemed to be as effective than Cti (fig 2 and 3). Conclusion This registry study gives a real life idea of the therapeutic intent in AML patients, stressing in particular the small proportion of patients who can be included in clinical trials due to comorbidity and also the small number of available clinical trials in elderly patients, due to the current lack of promising drug apart from IC (among pts>65y/o only 13 pts have been included). Our observatory shows that in pts >65 y/o the intent of treatment should take account MRC classification, CTI being more effective that DMT or LDAC only in intermediate cases. Disclosures No relevant conflicts of interest to declare.

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