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

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.

Study Overview

• Status: Recruiting
• Conditions: Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; High-risk Myelodysplastic Syndrome; Myeloproliferative Neoplasm, Unclassifiable; Secondary Myelofibrosis in Myeloproliferative Disease
• Intervention / Treatment: Other: Biobanking

• Study Type: Observational
• Enrollment (Estimated): 3000

Contacts and Locations

• Study Contact: Name: Jérôme Lambert, Pr; Phone Number: +33 142499742; Email: jerome.lambert@u-paris.fr
• Study Contact Backup: Name: Hervé Dombret, Pr; Phone Number: +33 1 57 27 68 47; Email: herve.dombret@aphp.fr

Study Locations

• France
  • Bobigny, France
    • Recruiting
    • Hopital Avicenne
    • Contact: Thorsten Braun, Pr; Email: thorsten.braun@aphp.fr
  • Paris, France
    • Recruiting
    • Hopital Saint Louis
    • Contact: Hervé Dombret, Pr; Phone Number: +33 1 57 27 68 47; Email: herve.dombret@aphp.fr
  • Paris, France
    • Recruiting
    • Hopital Robert Debre
    • Contact: André Baruchel, Pr; Email: andre.baruchel@aphp.fr

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

4

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
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; For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.
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; For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.
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; For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.
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; For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Overall Survival	at 5 years
Event Free Survival	at 5 years
Relapse Free Survival	at 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
Standardized evaluation of hematological response		at 5 years
Minimal measurable residual disease (MRD) response		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		After first consolidation cycle which is between 1 and 2 months
Minimal measurable residual disease (MRD) response		After last consolidation cycle which is between 3 and 8 months
Minimal measurable residual disease (MRD) response		Before HSCT
Minimal measurable residual disease (MRD) response		at day 100 after HSCT
Minimal measurable residual disease (MRD) response		at 5 years
Incidence of allogeneic HSCT		at 5 years
Modalities of allogeneic HSCT		at 5 years
Incidence of hematological relapses		at 5 years
Type of hematological relapses		at 5 years
Incidence of hematological progressions		at 5 years
Type of hematological progressions		at 5 years
Incidence of MRD relapses		at 5 years
Incidence of MRD progressions		at 5 years
Proportions of patients with treatment-related toxicities	Treatment-related toxicities will be Evaluated by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0	at 5 years
Cumulative incidences of relapse		at 5 years
Cumulative incidences of non-relapse mortality		at 5 years
Quality-of-life 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.	at inclusion
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	at the end of 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
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	after 2 consolidations courses which is between 3 months and 8 months
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	at 3 months after the end of treatment
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	at 6 months after the end of treatment
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	at 12 months after the end of treatment
Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire	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.	at day 100 after hematopoietic stem cell transplant
Incidence of secondary cancer		at 5 years
Incidence of secondary cancer		up to 15 years

Collaborators and Investigators

• Sponsor: Assistance Publique - Hôpitaux de Paris

Study record dates

Study Major Dates

• Study Start (Actual): March 28, 2022
• Primary Completion (Estimated): March 1, 2042
• Study Completion (Estimated): March 1, 2042

Study Registration Dates

• First Submitted: January 28, 2022
• First Submitted That Met QC Criteria: April 7, 2022
• First Posted (Actual): April 14, 2022

Study Record Updates

• Last Update Posted (Actual): May 29, 2024
• Last Update Submitted That Met QC Criteria: May 26, 2024
• Last Verified: May 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Leukemia, Lymphoid; Myelodysplastic Syndromes; Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myeloproliferative Disorders
• Other Study ID Numbers: APHP210850

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No