The Clinical Trials and Management of Acute Myeloid Leukemia

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Clinical Research of Cancer".

Deadline for manuscript submissions: 19 September 2025 | Viewed by 1641

Special Issue Editor


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Guest Editor
1. Department of Medical Oncology, Philadelphia, Thomas Jefferson University, Philadelphia, PA 19107, USA
2. Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA 19107, USA
Interests: acute myeloid leukemia; clinical trials; translational research; targeted therapies
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Special Issue Information

Dear Colleagues, 

Acute myeloid leukemia (AML) has always posed a therapeutic challenge due to its molecular and cytogenetic complexity. For the first time in decades, advances in treatment through the approval of novel agents and combinations, have been made. Long-term disease-free survival had plateaued at less than 30% at 5 years; however, in the last few years, a small, but steady increase has been seen thanks to the advancements in novel therapies. The exploration of novel mechanisms and immune system enhancement as therapeutic targets for the treatment of AML continues.

This Special Issue will highlight the clinical trials and advances in supportive care for acute myeloid leukemia. 

You may choose our Joint Special Issue in Current Oncology.

Dr. Margaret T. Kasner
Guest Editor

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Keywords

  • acute myeloid leukemia
  • clinical trials
  • translational research
  • targeted therapies
  • novel agents

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Published Papers (1 paper)

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Research

12 pages, 1748 KiB  
Article
A Phase I Trial of Sirolimus with “7&3” Induction Chemotherapy in Patients with Newly Diagnosed Acute Myeloid Leukemia
by Neil Palmisiano, Grace Jeschke, Lindsay Wilde, Onder Alpdogan, Matthew Carabasi, Joanne Filicko-O’Hara, Dolores Grosso, Thomas Klumpp, Ubaldo Martinez, John Wagner, Martin P. Carroll, Alexander Perl and Margaret Kasner
Cancers 2023, 15(21), 5129; https://doi.org/10.3390/cancers15215129 - 25 Oct 2023
Viewed by 1203
Abstract
Chemotherapy remains a primary treatment for younger AML patients, though many relapse. Data from our group have shown that highly phosphorylated S6 in blasts may predict response to sirolimus given with chemotherapy. We report the results of a phase I study of this [...] Read more.
Chemotherapy remains a primary treatment for younger AML patients, though many relapse. Data from our group have shown that highly phosphorylated S6 in blasts may predict response to sirolimus given with chemotherapy. We report the results of a phase I study of this combination in newly diagnosed AML and the pharmacodynamic analysis of pS6 before and after treatment. Subjects received sirolimus (12 mg on day 1, 4 mg daily, days 2–10), then idarubicin and cytarabine (days 4–10). Response was assessed at hematologic recovery or by day 42 using a modified IWG criteria. Fifty-five patients received sirolimus. Toxicity was similar to published 7 + 3 data, and 53% had high-, 27% intermediate-, and 20% favorable-risk disease. Forty-four percent of the high-risk patients entered into CR/CRp. Seventy-nine percent of the intermediate-risk subjects had a CR/CRp. All favorable-risk patients had a CR by day 42; 9/11 remained alive and in remission with a median follow-up of 660 days. Additionally, 41/55 patients had adequate samples for pharmacodynamic analysis. All patients demonstrated activation of S6 prior to therapy, in contrast to 67% seen in previous studies of relapsed AML. mTORC1 inhibition was observed in 66% of patients without enrichment among patients who achieved remission. We conclude that sirolimus and 7 + 3 is a well-tolerated and safe regimen. mTORC1 appears to be activated in almost all patients at diagnosis of AML. Inhibition of mTORC1 did not differ based on response, suggesting that AML cells may have redundant signaling pathways that regulate chemosensitivity in the presence of mTORC1 inhibition. Full article
(This article belongs to the Special Issue The Clinical Trials and Management of Acute Myeloid Leukemia)
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