The Patient Cohort of the National Center for Precision Medicine in Leukemia

Overview

If for years the treatment strategy of leukemia and related disorders (LRDs, including acute leukemias and predisposition syndromes) has been based solely on whether the patient could receive or not intensive chemotherapy and transplantation, the advent of new targeted or less targeted drugs has led to the development of a growing number of new therapeutic approaches, very often offered to specific patient/disease subsets, justifying the generic term of 'precision medicine'. As an international leukemia center of excellence, THEMA, the French National Center for Precision Medicine in Leukemia (selected as IHUB-2 by the French National Agency for Research), is a care, research, transfer and education initiative located at the Saint-Louis Research Institute (IRSL) in Paris and devoted to precision medicine in leukemia in a real-life environment. The present non-interventional study (eTHEMA) is a pillar of the whole THEMA project. As a prerequisite for precision medicine, this program focuses on individual data collection, aiming to collect high-quality data not only in patients treated into prospective clinical trials, but in every THEMA patient with a special interest in outpatients' care and research. The primary objective of this non-interventional study is to describe the baseline characteristics planned treatments and outcomes of patients newly diagnosed with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS), or myeloproliferative neoplasm (MPN)-related myelofibrosis, when managed and treated according to standard diagnosis and care practices.

Interventions

• Other Biobanking
  For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Primary outcome measures

• Event Free Survival [Time frame: at 5 years]
• Relapse Free Survival [Time frame: at 5 years]
• Overall Survival [Time frame: at 5 years]

Secondary outcome measures (12)

• Standardized evaluation of hematological response [Time frame: After induction cycle which is between between day 25 and day 42 for patients treated intensively and between Month 1 and Month 6 for patients treated treated with low intensity regimen]
• Standardized evaluation of hematological response [Time frame: After first consolidation cycle which is between 1 and 2 months]
• Standardized evaluation of hematological response [Time frame: After last consolidation cycle which is between 3 and 8 months]
• Standardized evaluation of hematological response [Time frame: Before HSCT]
• Standardized evaluation of hematological response [Time frame: at day 100 after HSCT]
• Standardized evaluation of hematological response [Time frame: at 5 years]
• Minimal measurable residual disease (MRD) response [Time frame: After induction which is between day 25 and day 42 for patients treated intensively and between month 1 and month 6 for patients with low intensity regimen]
• Minimal measurable residual disease (MRD) response [Time frame: After first consolidation cycle which is between 1 and 2 months]
• Minimal measurable residual disease (MRD) response [Time frame: After last consolidation cycle which is between 3 and 8 months]
• Minimal measurable residual disease (MRD) response [Time frame: Before HSCT]
• Minimal measurable residual disease (MRD) response [Time frame: at day 100 after HSCT]
• Minimal measurable residual disease (MRD) response [Time frame: at 5 years]

Eligibility criteria

Inclusion criteria

• Patient with newly diagnosed previously untreated de novo, secondary or therapy-related leukemia or related disorders (LRD), including AML, ALL, HR-MDS (according to the international score IPSS), and MNP-related myelofibrosis
• Patient informed and not opposed to participating
• Affiliation to social security or any health insurance

Exclusion criteria

• LRD which is not morphologically proven (patients with granulocytic sarcoma may be included)
• Previous treatment for LRD, apart from:

• Hydroxyurea or previous MDS/MPN-CML therapy in AML patients
• Steroids, vincristine, intrathecal prophylactic or curative injection or previous CML therapy in ALL patients
• Erythroid stimulating agents (ESAs), luspatercept, granulocyte colony-stimulating factor (G-CSF), eltrombopag or other TPO agonist, iron chelation therapy, hypomethylating agents (HMAs), lenalidomide or any investigational drug previously used to treat MDS in HR-MDS patients
• Hydroxyurea, standard or pegylated interferon alpha, ruxolitinib or other JAK inhibitors, busulfan, anagrelide, ESAs or any investigational drug previously used to treat MPN in MPN-related myelofibrosis patients
• Patient under guardianship / curatorship
• Patient under AME
• Opposition of the patient to be enrolled in the eTHEMA cohort

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

France · 3 centers

• Hôpital Avicenne — Bobigny
• Hopital Robert Debré — Paris
• Hôpital Saint Louis — Paris

Publications

• Zhao LP, Dumas-Rivero T, Barette L, Aguinaga L, Cheffai A, Chauvel C, Dal Bello R, Raffoux E, Clappier E, Duchmann M, Fenaux P, Lemaire P, Mathis S, Sebert M, Ades L, Itzykson R. Prognostic significance of monocytic-like phenotype in patients with AML treated with venetoclax and azacytidine. Blood Adv. 2025 Jul 22;9(14):3556-3565. doi: 10.1182/bloodadvances.2024015734. PMID 40249917

Identifiers

NCT: NCT05326919 · APHP210850

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗