Not all diseases - especially at an early stage - produce clear, characteristic symptoms that prompt patients to visit their doctor and allow the disease to be diagnosed at an early stage. Unfortunately, the same is true of some blood cancers, which can be confused with other ailments - in the case of older people, often with other chronic diseases. So insidious is mantle cell lymphoma.
Mantle cell lymphoma (MCL) is a malignant disease of the lymphatic system, diagnosed annually in Poland in approximately 300 patients, mainly aged 67-70 years. Unfortunately, the course is aggressive, relapses are frequent, periods of remission, i.e. withdrawal of symptoms of the disease, are becoming shorter and shorter, and prognosis is unfavourable. 5-6 years is the average survival time, but some patients die as early as six months after diagnosis.
Emergency access only
The standard therapeutic treatment is immunochemotherapy. However, resistance to this type of treatment may develop after the first relapse. What then remains? For today - only emergency access to drug technology. RDTL is unfortunately a prolongation in the process of receiving treatment. In Europe, access to drugs with a mechanism of action other than chemotherapy - Bruton's kinase inhibitors, such as ibrutinib therapy - has been the standard for many years.
Unfortunately, MCL patients in Poland cannot, for the time being, count on such a standard within the framework of a drug programme reimbursed by the National Health Fund, even though, according to the law, they should receive a treatment corresponding to current medical knowledge.
Why RDTL is not enough
RDTL is a system for issuing individual consents for the treatment of patients for whom all available options have been exhausted publicly funded therapies. As of 2019, more than 50 patients with MCL have qualified for RDTL treatment and 30 patients from the requested population are currently receiving the therapy.
However, the RDTL procedure should, as the name suggests, be lifesaving and serve in exceptional cases. The need to use it as a de facto standard procedure demonstrates that access to optimal treatment, which is already the norm in Europeshould be guaranteed by the state. Patients and the clinical community have been campaigning for an improvement in this situation, but the reimbursement process has been going on for more than three years and the drug has not been included in the next reimbursement list in July either.
- Patients who have received immunochemotherapy treatment and are refractory or with relapse after another line of immunochemotherapy virtually have no other effective treatment option, he stresses Prof Iwona Hus, Head of the Division of Lymphatic System Diseases at the Department of Haematology of the IHiT in Warsaw, President of the Polish Society of Haematologists and Transfusiologists. He points out that ibrutinib has been in all recommendations of international societies for several years. It should therefore also be a standard in Poland.
Sick people have no choice
Of course, doctors, out of concern for their patients, try to request treatment for their patients under RDTL. As Prof. I.Hus notes, these are indeed patients who have no other treatment option. The range of options is exhausted. The disease is unfortunately aggressive and relapses quickly, so it is important that access to therapy is as easy and quick as possible, so that withimprove the patient's chances for a longer life.
Meanwhile, the procedure RDTLDespite recent changes related to the Medical Fund Act, which have somewhat streamlined it, it is still time-consuming and the rules of financing raise concerns among some facilities. The centre settles accounts with the provincial branch of the NFZ on the basis of a bill or invoice documenting the purchase of a medicine and a positive opinion of a national or provincial consultant in a given field of medicine. This takes time, but also finances are not insignificant.
- RDTL treatment is still a question of the funds available to the facility. It is not easy. There is also the extra time and waiting for a decision. In a drug programme it would be much simpler -. assessed by Prof. Iwona Hus, haematologist.
Most patients with MCL at the time of diagnosis are already in the advanced stage of diseasewhich does not facilitate treatment. Currently, the therapy is already reimbursed in all other European countries. In 21 countries, the drug has been standard for more than four years, while in Poland the reimbursement process has already taken more than 1 000 days. This is one of the records.
Some facts about the MCL
The disease affects men three to four times more often than women. Like other lymphomasis caused by an error in the formation or maturation of immune cells, and the cause of its development is mostly unknown.
MCL shows features of both aggressive (rapid growth) and chronic (incurable) lymphomas. The disease has a persistently recurrent characterand as the disease progresses, periods of remission, i.e. withdrawal of symptoms, become shorter. The median overall survival is about 5-6 years. Some patients die as early as six months after diagnosis.
Unfortunately, it is difficult to recognise at an early stage due to scanty symptoms (sweats - especially night sweats, fevers and significant weight loss, often pruritic skin). Only one third of patients are symptomatic at the time of diagnosis. Even changes in the results of blood count do not always provide a diagnosis straight away. Diagnosis therefore takes a long time and most patients are already at an advanced stage of the disease, which does not facilitate treatment. The diagnosis usually includes enlarged lymph nodes, as well as liver, spleen and bone marrow involvement.
Source: politicshealth.com