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

Assistance Publique - Hôpitaux de Paris

Status: Enrolling

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

Treatments: Other: Biobanking

Study type: Observational

Funder types: Other

Identifiers

NCT05326919

APHP210850

Details and patient eligibility

About

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.

Enrollment: 3,000 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

3,000 participants in 4 patient groups

Acute myeloid Leukemia (AML)

Description:

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

Treatment:

Other: Biobanking

Acute lymphoblastic leukemia (ALL)

Description:

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

Treatment:

Other: Biobanking

High-risk myelodysplastic syndrome (MDS)

Description:

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

Treatment:

Other: Biobanking

Myeloproliferative neoplasm -related myelofibrosis

Description:

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

Treatment:

Other: Biobanking