International collaborative prospective study for children and adolescents with standard-risk ALK-positive anaplastic large-cell lymphoma (ALCL) to evaluate the efficacy of vinblastine (ALCL-VBL)

Anaplastic large cell lymphoma (ALCL) is a rare, aggressive form of lymphoma that primarily affects children and young adults. It belongs to the group of so-called non-Hodgkin lymphomas and originates from cells of the body’s immune system. ALCL develops when these cells (T lymphocytes) multiply uncontrollably and begin to displace the surrounding healthy tissue. Researchers have not yet determined what causes the cells to become malignant. However, they have identified several genetic changes that frequently occur in ALCL. These mutations can cause healthy cells to turn into cancer cells. In ALK-positive ALCL, the abnormal T cells have a characteristic mutation that causes them to produce a protein called “anaplastic lymphoma kinase” (ALK). This type of ALCL is then referred to as ALK-positive.

The standard treatment for ALK-positive ALCL consists of combination chemotherapy administered in several courses (cycles). Treatment is administered in the hospital as an infusion through a venous line. Although this treatment can achieve a permanent cure in 70–75% of children, it is also associated with side effects such as susceptibility to infection or an increased risk of secondary tumors over the course of a lifetime. The drug vinblastine is also a chemotherapeutic agent that has not yet been tested in clinical trials for use in newly diagnosed ALCL. However, vinblastine has already been shown to be highly effective in patients with a relapse (recurrence) of ALCL. The drug has also been approved for several years for the treatment of a wide variety of cancers.

The goal of the ALCL-VBL study is to achieve a cure rate of at least 75% through treatment with vinblastine alone (monotherapy) in patients with ALK-positive ALCL. In addition to the high cure rate, it is hoped that monotherapy will result in fewer side effects for patients.

Patients will receive vinblastine monotherapy over a period of 2 years. The drug is administered weekly via intravenous infusion for the first 18 months and every 2 weeks for the final 6 months. Treatment takes place in a day clinic (on an outpatient basis); hospitalization is necessary only in the event of complications. After treatment ends, further follow-up with regular checkups is planned for 1 year.

Children and adolescents under the age of 18 who have ALK-positive anaplastic large cell lymphoma (ALCL) are eligible to participate in the study. The ALCL must be classified as standard-risk and must not have been treated previously. An exception is a single administration (intrathecal administration) of a triple therapy (methotrexate, cytarabine, and prednisone or hydrocortisone) into the fluid surrounding the central nervous system (cerebrospinal fluid).