The Patient Cohort of the National Center for Precision Medicine in Leukemia
- Conditions
- Acute Lymphoblastic LeukemiaAcute Myeloid LeukemiaHigh-risk Myelodysplastic SyndromeSecondary Myelofibrosis in Myeloproliferative DiseaseMyeloproliferative Neoplasm, Unclassifiable
- Interventions
- Other: Biobanking
- Registration Number
- NCT05326919
- Lead Sponsor
- Assistance Publique - Hôpitaux de Paris
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 3000
- 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
-
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
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Acute myeloid Leukemia (AML) Biobanking Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored. Acute lymphoblastic leukemia (ALL) Biobanking Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored. Myeloproliferative neoplasm -related myelofibrosis Biobanking Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored. High-risk myelodysplastic syndrome (MDS) Biobanking Standard and routine care. For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.
- Primary Outcome Measures
Name Time Method Overall Survival at 5 years Event Free Survival at 5 years Relapse Free Survival at 5 years
- Secondary Outcome Measures
Name Time Method Incidence of secondary cancer up to 15 years Incidence of MRD progressions at 5 years Cumulative incidences of relapse at 5 years Standardized evaluation of hematological response at 5 years Minimal measurable residual disease (MRD) response at 5 years Incidence of MRD relapses at 5 years Modalities of allogeneic HSCT at 5 years Incidence of hematological relapses at 5 years Type of hematological progressions at 5 years Proportions of patients with treatment-related toxicities at 5 years Treatment-related toxicities will be Evaluated by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0
Incidence of allogeneic HSCT at 5 years Type of hematological relapses at 5 years Incidence of hematological progressions at 5 years Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire at day 100 after hematopoietic stem cell transplant Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.
Cumulative incidences of non-relapse mortality at 5 years
Trial Locations
- Locations (3)
Hopital Robert Debré
🇫🇷Paris, France
Hôpital Avicenne
🇫🇷Bobigny, France
Hôpital Saint Louis
🇫🇷Paris, France