Patients’ unmet needs beyond the success of treatment

For decades, being diagnosed with chronic myeloid leukaemia meant facing a disease with an often poor prognosis. Today, the situation has changed dramatically thanks to the advent of targeted therapies capable of blocking the mechanism that fuels the growth of leukaemia cells. As a result, the vast majority of patients are able to achieve long-term control of the disease and can look to the future with prospects that were once unthinkable. This revolution represents one of the most significant achievements of modern haematology. But when a disease becomes manageable for many years, the meaning of the word ‘cure’ also changes. It is no longer enough to ask whether a drug works or not; we must understand what it is like to live whilst taking it every day, often for an indefinite period, and sometimes for life. It is here that the unmet needs of people with chronic myeloid leukaemia come to the fore.

The first concerns tolerability: even highly effective medicines can cause side effects. Some potential side effects require specific monitoring and particular attention, especially in those living with other medical conditions. Others, whilst less severe in nature, can profoundly affect quality of life and daily routines. Symptoms such as tiredness, nausea, digestive problems, and muscle or joint pain, if persistent – occurring every day for years – can affect a person’s ability to work, their sleep, their social life and their freedom to organise their own day. This is why it is essential to listen to the patient and not to assess the treatment solely in terms of its effectiveness in controlling the disease. Two people with similar clinical outcomes may experience treatment very differently. Recognising a side effect promptly, trying to manage it and, where necessary, reassessing the treatment strategy means safeguarding not only the control of leukaemia but also quality of life. Tolerability is closely linked to a second issue: adherence, that is, taking medication regularly as prescribed by the specialist.

In chronic myeloid leukaemia, continuity of care is essential. Yet sticking to a daily treatment regime for years is not always easy. Side effects, practical difficulties, other treatments to be taken, or the psychological burden of a condition which, even when well-controlled and seemingly dormant, remains a part of the patient’s life may all come into play. When a patient struggles to adhere to their treatment, therefore, the response cannot simply be a call for discipline. What is needed is an open dialogue, in which it is possible to discuss even difficulties that may seem trivial. A relationship of trust with one’s doctor helps to find practical solutions and prevent interruptions or unauthorised changes to treatment, which can jeopardise the results achieved. Another challenge is personalisation. Not all people with chronic myeloid leukaemia have the same needs, and the best treatment choice is not necessarily the most potent treatment in the abstract, but rather the one that offers, for that individual, the best balance between efficacy, safety and long-term sustainability. Research is expanding the range of treatment options available to specialists.

Alongside the treatments that have transformed the history of the disease, new options with different mechanisms of action have emerged. Having alternatives means being able to tailor treatment more effectively when the response is not as expected or when side effects become difficult to manage. But it also means looking beyond the immediate control of leukaemia: today, wherever possible, the aim is to achieve ever deeper and more stable responses over time, without losing sight of the patient’s well-being. Then there is a goal that, until a few years ago, seemed almost unimaginable: for some patients, following a very deep and sustained response, it is possible to consider discontinuing treatment whilst continuing to monitor the disease closely. This is known as treatment-free remission. It is not a goal accessible to everyone and requires well-defined criteria, rigorous monitoring and a decision made in consultation with the haematologist. But it clearly illustrates the shift in perspective: not merely living a long life with leukaemia, but also, when conditions allow, reducing the burden of treatment. The challenge in the coming years will therefore be twofold: to continue improving disease control and to make life increasingly sustainable for those living with the condition. Because therapeutic success is not measured solely by laboratory results. It is also measured by the ability to work, nurture relationships, make plans and find fulfilment in one’s daily life. Treating chronic myeloid leukaemia today means balancing both these aspects.

*Specialist in Haematology, Researcher at the Department of Biomedicine and Prevention, University of Rome “Tor Vergata”