TFR results and resources

Guidelines and Recommendations on Treatment-Free Remission in CML 

First recommendations exist about how to manage to stop and re-start therapy, based on early data from previous studies like the French STIM studies, the largest stop study EUROSKI as well as company-sponsored studies ENESTop, ENESTfreedom and DASFREE. The ELN, NCCN (USA) and ESMO (Europe) have published first recommendations for stopping treatment outside the context of clinical trials and the institutional requirements of the centre and lab. However, there is still no wide consensus among the CML experts, prognostic factors for TFR success are not yet clear, and the emotional impact is not yet well understood by doctors and patients.

 

Our publication in LEUKEMIA

tfr 1Our paper “Treatment-free remission in chronic myeloid leukemia: the patient perspective and areas of unmet needs” has been published in the scientific journal Leukemia in 2020.

Our TFR for CML patients’ study shows patient-based evidence on unmet needs of CML patients during the treatment-free remission journey.

 

Open Access Article in Leukemia: https://www.nature.com/articles/s41375-020-0867-0

 

Our full slide deck with TFR4CML survey data

TFR PPT deck

Our ESHCML2020 Abstract and Oral Presentation

TFR4CML results presented at the European School of Hematology, 22nd Annual John Goldman Conference on Chronic Myeloid Leukemia: Biology and Therapy.

Our Abstract Chronic Myeloid Leukemia Patients’ Concerns during Phase I of the Treatment-Free Remission Journey: Communication Between Patients and Doctors in the Different CML Advocates Network Regions” was accepted to be presented by our co-founder Giora Sharf as part of the scientific session 7: #TreatmentFreeRemission - Predictive factors".

Read the full Abstract  pdf ESHCML 2020 Abstract CMLAdvocatesNetwork (348 KB)

 

Our IACH2020 Abstract and Poster Presentation

Abstract and Poster presented at the International Academy for Clinical Hematology (IACH) 2020 Congress.

Read the Poster  pdf IACH2020 Poster CMLAdvocatesNetwork (1.36 MB)

 

Our EHA 2020 Abstract and Poster

Our Abstract CML Patients’ Views on Psychological Support Throughout the Treatment-Free Remission Journey” has been accepted at the 25th Congress of the European Hematology Association starting June 11.

EHA 2020 Abstract high 

 

Due to the COVID-19 outbreak, this year #EHA2020 has become a virtual event as have many other congresses, and the EHA Virtual Congress Platform will be accessible until October 15, 2020.

You can check in detail the E-Poster presented in EHA25Virtual by clicking in the image below:

 

EHA25_Poster

 

ECRD online 1Our ECRD 2020 abstract and poster

 

We are happy to announce that the European Conference on Rare Diseases and Orphan Products 2020 has selected our abstract and poster "CML Patients’ Views on Psychological Support Throughout the Treatment-Free Remission Journey" to be presented at the conference that will take place online 14-15 May 2020.

TFR Poster ECRD 2020

 

The ECRD, organised by Eurodis and Orphanet, is recognised globally as the largest, patient-led rare disease event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape future rare disease policies.

 

Abstracts 1Our ASH 2019 Abstract

The 61st American Society of Hematology Congress 2019 had selected our abstract "CML Patients’ Views on Psychological Support throughout the Treatment-Free Remission Journey" published online in the November supplemental issue of the prestigious magazine BloodTo read the full text of the Abstract "CML Patients’ Views on Psychological Support throughout the Treatment-Free Remission Journey", please click on the image on the right.

 

Our ASH 2019 Poster

Our Abstract has been additionally selected for Poster presentation as part of the scientific session 632 Chronic Myeloid Leukemia: Therapy: Poster II. Hematology Disease Topics & Pathways: Adult, Diseases, CML, Study Population, Myeloid Malignancies, taking place on Sunday, December 8, 2019, 6:00 PM-8:00 PM at Hall B, Level 2 (Orange County Convention Center), Orlando, USA.

To check and download the #ASH19 Poster "Chronic Myeloid Leukemia Patients’ Views on Psychological Support Throughout the Treatment-Free Remission Journey", please, click on the image below:

Poster 1

 

 

The authors would like to thank the 122 member organizations of the CML Advocates Network for their assistance in promoting the #TFR4CML survey and the CML patients who participated in the TFR for CML patients research. 

 

 

 

News on TFR

In this section, we are collecting and publishing news focused on treatment-free remission in CML on a regular basis. We offer you to check the "News on TFR" section to be informed about latest advances in stopping treatment for CML patients.

 

Final 3-year Results of the Dasatinib Discontinuation Trial in Patients With Chronic Myeloid Leukemia Who Received Dasatinib as a Second-line Treatment

Okada, Masaya et al. Clinical Lymphoma, Myeloma and Leukemia , Volume 18 , Issue 5 , 353 - 360.e1 DOI:  https://doi.org/10.1016/j.clml.2018.03.004

Abstract


TFR study june articleSecond-generation tyrosine kinase inhibitors (TKIs), including dasatinib and nilotinib, are better at inhibiting BCR-ABL kinase activity than the first-generation TKI imatinib mesylate. Second-generation TKIs were introduced initially as a second-line treatment for the patients with disease resistant or who were intolerant to imatinib and resulted in a remarkable response rate.1 Because subsequent studies demonstrated that second-generation TKIs show superior efficacy to imatinib for newly diagnosed chronic myeloid leukemia (CML),2, 3 their use as a first-line treatment has increased.

Although accumulating evidence has shown that discontinuing imatinib is feasible, trials of patients receiving second-generation TKIs are lacking. Therefore, we conducted a phase II trial to investigate whether long-term TFR was achievable after discontinuing second- or subsequent-line dasatinib treatment after imatinib resistance or intolerance (the DADI trial [dasatinib discontinuation]).

We have confirmed the feasibility of dasatinib discontinuation in patients without imatinib-resistant disease who maintain MR4.0 for ≥ 1 year. Moreover, we have demonstrated that both the characteristics of leukemia cells (an intrinsic factor) and the host immunologic responses (an extrinsic factor) are involved in maintenance of TFR. These findings could be useful for identifying patients who will derive the greatest benefit                                                           from TKI discontinuation and enable optimization of the algorithm used for treatment of CML patients taking TKIs.

To read the full article please visit the Clinical Lymphoma, Myeloma, Leukemia Journal

 

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Considerations for Successful Treatment-free Remission in Chronic Myeloid Leukemia

Considerations for Successful Treatment-free Remission in Chronic Myeloid Leukemia. Goldberg, Stuart L. et al. Clinical Lymphoma, Myeloma and Leukemia , Volume 18 , Issue 2 , 98 – 105. DOI: https://doi.org/10.1016/j.clml.2017.11.006

TFR DOI 1Abstract

BCR-ABL1 tyrosine kinase inhibitors have dramatically improved outcomes for patients with chronic myeloid leukemia, and current studies are investigating whether some patients may be able to suspend therapy yet maintain response in a state known as “treatment-free remission” (TFR). Results from ongoing studies suggest that ≈ 40% to 60% of patients in sustained (generally ≥ 2 years) deep molecular response (defined as a 4-log or deeper reduction in BCR-ABL1 transcripts, depending on the study) who attempt TFR may successfully remain off treatment. Results from TFR clinical trials, patient considerations for attempting TFR, and potential predictive factors associated with successful TFR are reviewed herein.

To read the full article please visit the Clinical Lymphoma, Myeloma, Leukemia Journal

 

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fdalogoFDA updates the label of Tasigna to reflect that certain patients with a type of leukaemia that may be eligible to stop treatment after sustained response

Discontinuation in treatment marks a first in Chronic Myeloid Leukemia

FDA News Release. December 22, 2017.

The U.S. Food and Drug Administration has updated the product label for the cancer drug Tasigna (nilotinib) to include information for providers about how to discontinue the drug in certain patients. Tasigna, first approved by the FDA in 2007, is indicated for the treatment of patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukaemia (CML). With today’s updated dosing recommendations, patients with early (chronic) phase CML who have been taking Tasigna for three years or more, and whose leukaemia has responded to treatment according to specific criteria as detected by a test that has received FDA marketing authorization, may be eligible to stop taking Tasigna.

“Patients diagnosed with CML generally face a lifetime of treatment to keep their leukaemia from growing or recurring,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval shows that some patients may be able to stop treatment with Tasigna altogether if they are showing a strong response to therapy. While we welcome this progress in patient care, it’s important to note that any discontinuation of treatment still means patients must be regularly monitored for disease recurrence.”

To read more, please visit the U.S. Food and Drug Administration website

 

 

TFR 4 CML PATIENTS

  Treatment free remission

 

Treatment free remission in CML

The advent of TKIs into the management of patients with CML has a profoundly improved the prognosis of CML patients, with a survival under continued therapy to be close to that of the normal population.

Following a lot of public and professional debate about previous and ongoing STOP trials in CML, stopping CML treatment has become a widely debated topic. According to current first-line studies (ENEST1st, ENESTnd, DASISION), about 65-75% of CML patients will, over years, achieve a durable deep molecular response (minimum MR4) on current TKI treatments. Of those who then stop treatment over some years, about half of them seem to remain in durable therapy-free remission.

Therefore, Treatment Free Remission (TFR) in CML is referred to having a stable deep molecular response without the need for ongoing Tyrosine Kinase Inhibitor (TKI) treatment after having been in deep molecular residual disease over a longer period of time. First recommendations exist about how to manage to stop and re-start therapy, based on early data from previous studies like the French STIM studies, the largest stop study EUROSKI as well as company-sponsored studies ENESTop, ENESTfreedom and DASFREE. The ELN, NCCN (USA) and ESMO (Europe) have published first recommendations for stopping treatment outside the context of clinical trials and the institutional requirements of the centre and lab. However, there is still no wide consensus among the CML experts, prognostic factors for TFR success are not yet clear, and the emotional impact is not yet well understood by doctors and patients.

 

TFR 4 CML PATIENTS Survey: Results

Leukemia Journal article

TFR PublicationThe CML Advocates Network has undertaken a global project focused on TFR with 1016 participants, because this topic is being debated by doctors, patients, regulators and pharmaceutical industry for different reasons, and the patient community feels this also needs careful, considerate communication in all channels based on the patients perspective. The “TFR 4 CML PATIENTS” project ran of a global online survey of TFR patients to collect the viewpoints, needs and experiences of CML patients that are considering or have already started Treatment Free Remission (TFR). The aim was to provide CML patients and healthcare providers with more evidence when considering or managing TFR.

The results have been published in the Journal Treatment-free remission in chronic myeloid leukemia: the patient perspective and areas of unmet needs. Sharf, Marin, Bradley, Pemberton-Whiteley, Bombaci, Christensen, Gouimi, Deekes, Daban, Geissler, in: Leukemia (2020), doi: 10.1038/s41375-020-0867-0

Full PowerPoint Slide deck with results

TFR PPT deckA full PowerPoint slide deck with the main findings can be downloaded here.

Results were also presented in an oral session of the ESH 2020 meeting. Posters were presented at EHA 2020 and ASH 2020.

Posters and additional resources

For additional posters  about our survey as presented at ASH, EHA and ESH, see our TFR Results and Resources page.

Project information

For more information about the TFR 4 CML PATIENTS survey, visit “TFR 4 CML PATIENTS SURVEY” webpage.

 

 

 

 

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