ArticleBlood2010
Shortcomings in the clinical evaluation of new drugs: acute myeloid leukemia as paradigm.
Article in Blood, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 37 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
37 citing papers in PubMed.
- Intergroup LEAP trial (S1612): A randomized phase 2/3 platform trial to test novel therapeutics in medically less fit older adults with acute myeloid leukemia.American journal of hematology · 2018Trial
- Trial
- Outpatient bendamustine and idarubicin for upfront therapy of elderly acute myeloid leukaemia/myelodysplastic syndrome: a phase I/II study using an innovative statistical design.British journal of haematology · 2014Trial
- Phase II trial of vorinostat and gemtuzumab ozogamicin as induction and post-remission therapy in older adults with previously untreated acute myeloid leukemia.Haematologica · 2012Trial
- Nigericin-induced apoptosis in acute myeloid leukemia via mitochondrial dysfunction and oxidative stress.Oncology research · 2025Article
- Article
- Stratified randomization for platform trials with differing experimental arm eligibility.Clinical trials (London, England) · 2021Article
- Level of evidence for FDA drug approvals in pivotal clinical trials of hematological malignancies.Leukemia & lymphoma · 2021Article
- Two decades of targeted therapies in acute myeloid leukemia.Leukemia · 2021Review
- Integrative Review of Managed Entry Agreements: Chances and Limitations.PharmacoEconomics · 2020Review
- Curcumin Combined with Thalidomide Reduces Expression ofDrug design, development and therapy · 2020Article
- HLA class I-restricted T cell epitopes isolated and identified from myeloid leukemia cells.Scientific reports · 2019Article
- Article
- Review
- Future prospects of therapeutic clinical trials in acute myeloid leukemia.Future oncology (London, England) · 2017Review
- Acute myeloid leukemia therapy and the chosen people.Leukemia · 2017Article
- Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel.Blood · 2017Review
- Curcumin Veto the Effects of Osteopontin (OPN) Specific Inhibitor on Leukemic Stem Cell Colony Forming Potential via Promotion of OPN Overexpression.International journal of hematology-oncology and stem cell research · 2016Article
- Phase I Clinical Trials in Acute Myeloid Leukemia: 23-Year Experience From Cancer Therapy Evaluation Program of the National Cancer Institute.Journal of the National Cancer Institute · 2016Article
- In silico model-based inference: an emerging approach for inverse problems in engineering better medicines.Current opinion in chemical engineering · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
Abstract
Drugs introduced over the past 25 years have benefitted many patients with acute myeloid leukemia (AML) and provided cure for some. Still, AML remains difficult to treat, and most patients will eventually die from their disease. Therefore, novel drugs and drug combinations are under intense investigation, and promising results eagerly awaited and embraced. However, drug development is lengthy and costs are staggering. While the phase 1-phase 2-phase 3 sequence of clinical drug testing has remained inviolate for decades, it appears intrinsically inefficient, and scientific flaws have been noted by many authors. Of major concern is the high frequency of false-positive results obtained in phase 2 studies. Here, we review features of phase 2 trials in AML that may contribute to this problem, particularly lack of control groups, patient heterogeneity, selection bias, and choice of end points. Recognizing these problems and challenges should provide us with opportunities to make drug development more efficient and less costly. We also suggest strategies for trial design improvement. Although our focus is on the treatment of AML, the principles that we highlight should be broadly applicable to the evaluation of new treatments for a variety of diseases.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.