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

NCT05326919 | Recruiting

• 1 other identifier: APHP210850
• Study Type: observational
• Target: 3,000
• Locations: 1 country
• Sites: 3

Brief Summary

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.

Conditions

Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; High-risk Myelodysplastic Syndrome; Secondary Myelofibrosis in Myeloproliferative Disease; Myeloproliferative Neoplasm, Unclassifiable

Outcome Measures

Primary Outcomes (3)

• Event Free Survival

  at 5 years

• Relapse Free Survival

  at 5 years

• Overall Survival

  at 5 years

Secondary Outcomes (32)

• Standardized evaluation of hematological response

  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

  After first consolidation cycle which is between 1 and 2 months

• Standardized evaluation of hematological response

  After last consolidation cycle which is between 3 and 8 months

• Standardized evaluation of hematological response

  Before HSCT

• Standardized evaluation of hematological response

  at day 100 after HSCT

Study Arms (4)

Acute myeloid Leukemia (AML)

Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Other: Biobanking

Acute lymphoblastic leukemia (ALL)

Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Other: Biobanking

High-risk myelodysplastic syndrome (MDS)

Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Other: Biobanking

Myeloproliferative neoplasm -related myelofibrosis

Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Other: Biobanking

Interventions

Biobanking OTHER

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

Acute lymphoblastic leukemia (ALL); Acute myeloid Leukemia (AML); High-risk myelodysplastic syndrome (MDS); Myeloproliferative neoplasm -related myelofibrosis

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with newly diagnosed 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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (3)

Hôpital Avicenne

Bobigny, France

RECRUITING

Hopital Robert Debré

Paris, France

RECRUITING

Hôpital Saint Louis

Paris, France

RECRUITING

Related Publications (1)

• 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 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myeloproliferative Disorders

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Leukemia, Lymphoid; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Bone Marrow Diseases

Central Study Contacts

Hervé DOMBRET, Pr

CONTACT

1 57 27 68 47; herve.dombret@aphp.fr

Jérôme Lambert, Pr

CONTACT

142499742; jerome.lambert@u-paris.fr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2022
• First Posted: April 14, 2022
• Study Start: March 28, 2022
• Primary Completion (Estimated): March 1, 2042
• Study Completion (Estimated): March 1, 2042
• Last Updated: May 29, 2024

Record last verified: 2024-05

Locations

FR (3)