Novartis Presents New Real World Evidence Data at ASH from the TARGET UK CML Study Comparing Current UK Clinical Practice with Existing European LeukemiaNet (ELN) 2013 Guidelines

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Novartis UK

11 Dec, 2017, 07:00 GMT

CAMBERLEY, England, December 11, 2017 /PRNewswire/ --

  • Interim data provides important real world insights into the treatment pathway of patients in the UK with chronic myeloid leukaemia   
  • In patients with a suboptimal response in first line; higher rates of subsequent ELN optimal response were observed in patients who switched therapy[1]
  • Results provide evidence that optimal ELN molecular responses and deep molecular responses can also be achieved in patients with prior resistance to first line treatment[1]

Novartis today announced interim results from the TARGET UK study, an ongoing study to evaluate the treatment of UK patients with Chronic Myeloid Leukaemia (CML), compared to the European LeukemiaNet 2013 (ELN2013) recommendations for CML management.[1],[2] These findings, presented at 59th American Society of Hematology (ASH) Annual Meeting & Exposition, Atlanta, December 9-12, indicate that although management of UK patients was largely in line with ELN2013 recommendations, there were areas in which they could be better applied.[1]

The TARGET UK study is an observational, retrospective study being conducted at UK NHS secondary/tertiary care centres. This ongoing study aims to evaluate tyrosine kinase inhibitor (TKI) treatment pathways, monitoring patterns and real-world response rates in UK patients with CML against ELN2013 recommendations. Interim results for the first 126 recruited patients from 14 UK centres were presented today.

Data on patient characteristics, TKI choice and molecular responses were collected by a review of medical records for adult patients (≥18 years) with chronic phase CML, median follow-up of 21.5 (range 13.6 to 28.0) months.[1] Median age at first TKI was 53.7 (range 37.6 to 65.7) years and 52% of patients were male.[1]

The ELN2013 recommendations for CML management have an increased focus on monitoring of molecular responses, describing "optimal", "warning" and "failure" at specific milestones in response to treatment with TKIs to guide changes in therapy.[2] Interim findings from the TARGET UK study reveal that patients who switched TKI following an ELN "warning" or "failure" had a higher observed rate of optimal ELN response than those who remained on first line therapy despite an ELN "warning" or "failure" response.[1] Overall, the frequency of molecular response testing observed in the interim analysis of TARGET was in line with ELN recommendations with a majority of patients having three or more PCR BCR-ABL tests in the first year of treatments.[1]

"The TARGET UK study is an important milestone in CML research as it provides us with real-world UK data which has previously been lacking," said Professor Adam Mead, Associate Professor of Haematology, Weatherall Institute of Molecular Medicine (WIMM), University of Oxford. "This interim analysis supports the use of the ELN guidelines in the UK as well as providing new insights into the speed and depth of response to TKI treatment both of which are known to be important indicators of patient outcomes in CML."

Frequency and timing of molecular assessments and switching TKI therapy in the event of an ELN "failure" were highlighted as areas in which the ELN2013 recommendations could be better applied.[1] The data also highlight that optimal ELN responses and deep molecular response can be achieved in patients with prior resistance to a first line TKI treatment, further supporting switching TKI therapy under certain conditions.[1] Furthermore, the study demonstrates in a real world setting that a higher proportion of patients treated with a second generation TKI (either first or second line) achieve major molecular response and deeper molecular responses than those patients treated with imatinib.[1]

Barak Palatchi, Oncology General Manager, Novartis UK & Ireland, said: "Treatment developments in CML have significantly improved outcomes for the majority of patients. This real-world data is key to our ongoing understanding in this area, helping to support further improvements in clinical practice."

An early molecular response (EMR; BCR-ABL1 IS <10%) at three months and major molecular response (MMR; BCR-ABL1 IS ≤0.1%) within 12 months are defined as key optimal responses by the ELN.[2] More recently, the achievement of a deep response of MR4.5 (BCR-ABL1 IS <0.0032%) has also been associated with improved outcomes[3] and a sustained deep molecular response is an important requirement for treatment-free remission.[4],[5] Real world UK data focused on these outcomes are currently lacking, particularly in patients switching to second line treatment.

Further recruitment and extended follow-up are planned as part of the TARGET UK study in 21 centres. This will enable more detailed analyses of UK real world monitoring and CML management patterns compared to the ELN2013 recommendations.

About Tasigna (nilotinib)   

Tasigna® (nilotinib) is approved in more than 122 countries for the treatment of chronic phase and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukaemia (Ph+ CML) in adult patients resistant or intolerant to at least one prior therapy, including Glivec® (imatinib), and in more than 110 countries for the treatment of adult patients with newly diagnosed Ph+ CML in chronic phase.

Novartis Commitment to CML   

Over the past several decades, Novartis research in Ph+ CML has helped transform the disease from a fatal leukaemia to a chronic condition in most patients, and today, the company continues its long-standing commitment to the global CML community. Novartis follows the science and builds upon existing evidence to explore what could be the next major contribution in the treatment of Ph+ CML. The company is evaluating more than 1,000 patients as part of the Tasigna ENEST studies, which include ENESTfreedom and ENESTop as well as two other ongoing company-sponsored TFR studies and multiple investigator-initiated studies, as well as investigational compounds.

About Novartis  

Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 121,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.

In the UK, Novartis develops, manufactures and markets innovative medicines, devices and diagnostic tests which help improve patient outcomes. Based on four sites across the north and south of England, we employ approximately 1,500 people to serve healthcare needs across the whole of the UK, as well as supporting the global operations of Novartis by manufacturing the active pharmaceutical ingredients used worldwide in many medicines. In 2016 Novartis in the UK invested almost £40million in R&D and is the largest commercial sponsor of clinical trials. For more information, please visit http://www.novartis.co.uk.

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References  

  1. Milojkovic, D. et al. The Target UK study: Real-World Evidence of Molecular Response to Tyrosine Kinase Inhibitors Supports European LeukemiaNet 2013 Recommendations for the Management of Chronic Myeloid Leukaemia. Poster presentation. Abstract #2892. Annual Meeting of the American Society of Hematology, 2017.
  2. Baccarani et al. 2013. European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013. Blood. 122(6): 872-884.
  3. Hehlmann et al. 2014. Deep Molecular Response Is Reached by the Majority of Patients Treated With Imatinib, Predicts Survival, and Is Achieved More Quickly by Optimized High-Dose Imatinib: Results From the Randomized CML-Study IV. J Clin Oncol. 32(5): 415-23.
  4. NCCN Clinical Practice Guidelines® in Oncology (NCCN Guidelines®) Chronic Myeloid Leukaemia. (version 1.2018) Published 10.31.2017.
  5. Hochhaus, A. et al. 2017. Chronic myeloid leukaemia: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Annals of Oncology. 28 (Supplement 4): iv41-iv51.

ONC17-E062  Date of preparation: December 2017

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