All content on this site is intended for healthcare professionals only. By acknowledging this message and accessing the information on this website you are confirming that you are a Healthcare Professional. If you are a patient or carer, please visit Know AML.

The AML Hub uses cookies on this website. They help us give you the best online experience. By continuing to use our website without changing your cookie settings, you agree to our use of cookies in accordance with our updated Cookie Policy


Now you can personalise
your AML Hub experience!

Bookmark content to read later

Select your specific areas of interest

View content recommended for you

Find out more

The AML Hub website uses a third-party service provided by Google that dynamically translates web content. Translations are machine generated, so may not be an exact or complete translation, and the AML Hub cannot guarantee the accuracy of translated content. The AML Hub and its employees will not be liable for any direct, indirect, or consequential damages (even if foreseeable) resulting from use of the Google Translate feature. For further support with Google Translate, visit Google Translate Help.

Steering CommitteeAbout UsNewsletterContact
You're logged in! Click here any time to manage your account or log out.
You're logged in! Click here any time to manage your account or log out.

Ivosidenib for patients with IDH1 mutated R/R AML after allo-HSCT

Apr 19, 2024
Learning objective: After reading this article, learners will be able to cite a new development in the treatment of acute myeloid leukemia.

Bookmark this article

Targeted therapies are often a therapeutic option for patients with acute myeloid leukemia (AML) who are not eligible for intensive chemotherapy regimens or allogeneic hematopoietic stem cell transplantation (allo-HSCT), and for patients with relapsed/refractory AML (R/R AML).1 Although ivosidenib, an IDH1 inhibitor, has shown efficacy in both newly diagnosed and R/R AML, there is limited data on ivosidenib in patients with R/R AML after allo-HSCT.1

Recently, Caillet et al.1 published results from the IDALLO study in Hemasphere, investigating the efficacy and safety of ivosidenib in patients with IDH1-mutated R/R AML post-allo-HSCT. We summarize the key findings below.

Study design1

  • A multicenter retrospective study that enrolled patients with IDH1-mutated AML in relapse post-allo-HSCT, between June 2018 and April 2021.
  • Data was collected through the European Bone Marrow Transplantation Registry.
  • Ivosidenib was administered at 500 mg, once daily as a single agent.
  • The study endpoints were overall response rate (complete response and complete response with incomplete hematologic recovery), overall survival, progression-free survival, and safety.

Key findings1

  • A total of 22 patients were included in the study with a median age of 54.9 years.
  • The median follow-up period was 27.4 months.
  • The median time to relapse after transplant was 5.8 months.
  • Results for the survival endpoints are shown in Table 1.

Table 1. Study survival endpoints for patients with R/R AML treated with ivosidenib after allo-HSCT*

Survival endpoints

N = 22

ORR, %


CR, %


Median time to response, days


Median DOR, months


Median OS,  months


Not reached



Median PFS, months


Not reached



CR, complete response; CRi, CR with incomplete hematological recovery; DOR, duration of response; OS, overall survival; ORR, overall response rate; PFS, progression-free survival.

*Data from Caillet, et al.1

ORR is defined by CR + CRi.

Median OS in responders versus non-responders was the only statistically significant difference (p < 0.001).

  • At ivosidenib initiation, 45% of patients experienced neutropenia, and 55% experienced thrombocytopenia.
  • After ivosidenib initiation, 23% of patients experienced Grade 3–4 neutropenia.
  • In total, 14% of patients experienced an invasive fungal infection.
  • Overall, 77% of patients discontinued treatment during the follow-up period
    • No treatment discontinuations were due to toxicity.
  • 36% of patients died during the follow-up period
    • The main cause of death was disease progression.

Key learnings 

  • This retrospective study supports the efficacy and favorable safety profile of ivosidenib as a single agent therapy in R/R AML after allo-HSCT.
  • Further investigation is required to identify which patients are most likely to respond.
  • Several studies evaluating ivosidenib in combination with other treatments in the posttransplant setting as a maintenance therapy are ongoing.

  1. Caillet A, Simonet-Boissard M, Forcade E, et al. IDALLO study: A retrospective multicenter study of the SFGM‐TC evaluating the efficacy and safety of ivosidenib in relapsed IDH1‐mutated AML after allogeneic hematopoietic cell transplantation. Hemasphere. 2024;8(3):e DOI: 10.1002/hem3.44


Subscribe to get the best content related to AML delivered to your inbox