The Doctor Balmis University General Hospital in Alicante will participate in an international clinical trial seeking to improve the treatment of myelofibrosis, a rare type of blood cancer that affects the bone marrow. Blood cancers are the fifth most common cancer in the population. Myelofibrosis is a type of neoplasm in which the bone marrow is replaced by scar tissue (fibrosis). It is classified as a form of chronic leukaemia and carries up to a 20% risk of progressing to acute myeloid leukaemia.
The study, sponsored by the pharmaceutical company Novartis, was authorised in Spain on June 1st and is still in its initial phase, with patient recruitment not yet underway. It is a Phase III clinical trial—the most advanced stage before a drug can be approved—in which nearly 300 patients will participate internationally.
Myelofibrosis is a rare disease in which the bone marrow, responsible for producing blood cells, is gradually replaced by fibrous tissue. This causes symptoms such as anaemia, intense fatigue, and an enlarged spleen, which can significantly affect patients’ quality of life.
Rare disease
Because it is a rare disease, therapeutic advances are usually slower, so the launch of new clinical trials is key.
The trial will evaluate the effectiveness of combining two medications : ruxolitinib, a treatment already used for this disease, and pelabresib, an investigational drug. The main objective is to determine whether this combination improves current outcomes by reducing spleen size and alleviating symptoms after 24 weeks of treatment.
In addition, the researchers will analyse other relevant aspects such as patient survival, disease progression, and its impact on quality of life.
Hematology
In Spain, the study will also include the participation of the Virgen de la Arrixaca hospital in Murcia, the Salamanca hospital and the León and El Bierzo health area, as well as other reference hospitals in haematology such as the Hospital Clinic of Barcelona, the Hospital Universitari Vall d’Hebron, the Gregorio Marañón University General Hospital in Madrid and the University Hospital of Navarra.
Also included are the Donostia University Hospital, the Ramón y Cajal University Hospital, the Regional University Hospital of Málaga, the Quirónsalud Madrid University Hospital and the Catalan Institute of Oncology (Girona and L’Hospitalet).
The involvement of such diverse centres places several communities within an international network of research in haematological diseases, facilitating patient access to innovative therapies that are not yet generally available.
More affected
The fact that the study is in phase III indicates that there is already prior data that supports its potential, although it is still necessary to confirm its efficacy and safety in a larger number of patients.
At the moment, the trial has not yet begun to include participants, so the hospitals involved will soon begin preparations to select patients who meet the established criteria.
Although we will have to wait for the results, this type of research represents another step towards more effective and personalised treatments, especially in the field of rare diseases, where every advance can make a significant difference.
