the most frequent type of cancer in pediatric age, affects mainly between 3 and 5 years, and 350-400 new cases are diagnosed each year in our country. Acute lymphoblastic leukemia is a rare disease that progresses rapidly and can lead to death within days or weeks if left untreated. Fortunately, there are several effective therapies that allow healing, but when relapses occur, the situation is much more complicated. Now data from a study published in the scientific journal JAMA show for an important step forward: survival without worsening of the disease is longer than chemotherapy in young patients treated with a new immunotherapy drug. In fact, after almost two years, 69% of patients treated with the drug blinatumomab live and free from cancer compared to 43% of patients treated with chemotherapy a survival.

Particularly aggressive form that affects children and adults

80% of children with acute lymphoblastic leukemia recover, but relapses of this tumor are very resistant to treatment and, unfortunately, only 30% of them survive a relapse. Recently it was approved in Italy (also for adults). ” use of the new immunotherapy blinatumumab, which very effectively fights minimal residual disease, i.e. those cancer cells invisible to optical microscope checks which, after treatment, can remain even when the disease no longer gives symptoms and the patient’s parameters have returned normal, thus increasing the risk of relapse. Acute lymphoblastic leukemia from precursors of B cells is a particularly aggressive form that affects children and adults – explains Franco Locatelli, director of the Department of Onco-Hematology and Cell and Gene Therapy at the RCCS “Bambino Ges” Pediatric Hospital in Rome -. Unfortunately, about 15% of young patients relapse after a first line of chemotherapy. The earlier the relapse, the lower the chances of recovery and that a new course of treatment will be successful. On the contrary, in the absence of relapse, the hopes of definitive recovery are greater, as are the chances of being candidates for transplantation for patients who need it. For this reason, we need new options in this specific area. It is in this context that the results of the phase three trial, published in the latest issue of the JAMA (Journal of the American Medical Association), which evaluated the efficacy, safety and tolerability of blinatumomab compared to consolidation chemotherapy prior to allogeneic haematopoietic stem cell transplantation in pediatric patients with B-cell precursor acute lymphoblastic leukemia who have developed a first high-risk disease relapse.

Experimentation: more effective and less toxic

The trial enrolled 108 children, divided in half into two groups. The new immunotherapy drug has shown a longer survival without worsening of the disease than chemotherapy. Furthermore, among the patients who had a residual tumor cell at the start of the study, potentially capable of giving rise to disease recurrence (minimal residual disease), 93% reached the state of negativity (i.e. there were no more traces of the tumor) after treatment with immunotherapy, compared with 24% of patients undergoing chemotherapy. The three-year overall survival estimate in the blinatumomab group was 81% versus 56% in those who received chemo. The incidence of serious adverse events (none fatal) was 24.1% and 43.1%, respectively .I am very satisfied that the study results demonstrated greater efficacy of blinatumomab compared to intensive chemotherapy as well as a lower incidence of severe toxicity – adds Locatelli, professor of paediatrics at the La Sapienza University of Rome and coordinator of the European phase III study, which in Italy involved about 50 small patients in eight centers -. Chemotherapy has so far been used as the primary consolidation treatment for all patients in first relapse of the disease with high-risk characteristics before stem cell transplantation: this approach is only partially effective and associated with an important toxicity. Blinatumomab has proven to be a safer and more effective consolidation therapy option for pediatric patients – we have an additional therapeutic tool to heal this subset of children.


At the same time as the results of the European study, the results of a Phase 3 trial by the Children’s Oncology Group, which involved a further 208 patients: children, adolescents and young adults (up to the age of 30), were also published in JAMA. ) with B-cell precursor acute lymphoblastic leukemia at first relapse were treated with blinatumomab or chemotherapy before undergoing allogeneic stem cell transplantation. In this study, too, treatment with blinatumomab demonstrated a better chance of event-free survival, a reduction in severe toxicity, a higher rate of negativization of minimal residual disease, a greater chance of allogeneic haematopoietic stem cell transplantation, and a better overall survival than chemotherapy.

March 23, 2021 (change March 23, 2021 | 17:53)

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