What the trial was testing
The trial enrolled 76 patients with leukemia. The study was sponsored by Daiichi Sankyo and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Thirty percent of patients with relapsed blood cancer went into remission on this daily pill.
Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2013 · NCT00462761
These findings — that went into remission after previous treatments had failed — were published in the Journal of clinical oncology : official journal of the American Society of Clinical Oncology and represent the headline result of the study.
Researchers tracked outcomes across 76 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with leukemia, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
This treatment showed promise for people whose blood cancer had returned or wasn't responding to other therapies, especially those with a specific genetic change. This was an early safety study and the pill is not yet FDA-approved. Ask your doctor about open trials or related approved options for relapsed blood cancer.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open leukemia trials
Treatment Protocol for Children and Adolescents With Acute Lymphoblastic Leukemia - AIEOP-BFM ALL 2017
The understanding of acute lymphoblastic leukemia (ALL) in childhood and adolescence has largely changed due to extensive genetic research in recent years: ALL is now considered to be a very heterogeneous disease group. The leukemia cells present themselves with quite differently activated regulatory mechanisms of the malignant phenotype. The introduction of more accurate methods of assessing therapy response ("minimal residual disease \[MRD\] tests") has provided new insights into very different mechanisms of action, including factors influenced by host factors; this has had practical clinical consequences for the use of more individualized therapy. Multimodal therapies have enabled a cure level of over 80% for ALL in this age group. However, the own and international study data show that the therapy toxicity of the contemporary chemotherapy concepts has become unacceptably high, in particular with respect to those intensified therapies used for the treatment of patients at high risk of ALL relapse. The AIEOP-BFM ALL 2017 study therefore aims for an innovative integrated approach that will not only adapt the risk stratification to new prognostic markers using more comprehensive diagnostics, but above all, qualitatively reorient the therapy. The most important consequence will be that this study is testing immunotherapy with the bispecific antibody blinatumomab as an alternative to particularly intensive and toxic chemotherapy elements in precursor B-cell ALL (pB-ALL) patients with detectable chemotherapy resistance and at high risk of relapse. With the aim to complement the effects of the conventional chemotherapy, Blinatumomab is in addition tested in the large group of pB-ALL patients at intermediate relapse risk with seemingly unremarkable leukemia, but who account for a large proportion of all relapses. Targeted therapy is also used in the form of the proteasome inhibitor bortezomib for patients with pB-ALL and slow response to the drugs of the induction chemotherapy with the aim to overcome intrinsic chemotherapy resistance of the ALL cells. In patients with T-lineage ALL, who have particularly poor chances for cure after relapse, the established consolidation chemotherapy has proved to be particularly effective. This chemotherapy phase is therefore tested in a longer and more intensive form in such T-ALL patients with intermediate or slow early treatment response with the aim to reduce the relapses rate in this subgroup.
Post-authorization Safety Study of Allogeneic Hematopoietic Stem Cell Transplantation in Patients Treated With Mogamulizumab
This is a non-interventional cohort study evaluating non-relapse mortality and toxicities in patients with CTCL or ATLL treated with mogamulizumab pre- or post- alloHCT for patients transplanted beginning January 1, 2012 until accrual is complete.