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NCT Number: NCT05326919

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

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.

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Key information

Conditions

Acute Myeloid Leukemia Acute Lymphoblastic Leukemia Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases High-risk Myelodysplastic Syndrome Immune System Diseases Immunoproliferative Disorders Leukemia Leukemia, Lymphoid Leukemia, Myeloid Leukemia, Myeloid, Acute Lymphatic Diseases Lymphoproliferative Disorders Myeloproliferative Disorders Myeloproliferative Neoplasm, Unclassifiable Neoplasms Neoplasms by Histologic Type Precursor Cell Lymphoblastic Leukemia-Lymphoma Secondary Myelofibrosis in Myeloproliferative Disease

Sex eligibility

All sexes

Study type

Observational

Primary location

Hôpital Avicenne, Bobigny, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Biobanking

Other

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

Primary outcomes

  1. Event Free Survival

    Time frame: at 5 years

  2. Relapse Free Survival

    Time frame: at 5 years

  3. Overall Survival

    Time frame: at 5 years

Secondary outcomes

  1. 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

  2. Standardized evaluation of hematological response

    Time frame: After first consolidation cycle which is between 1 and 2 months

  3. Standardized evaluation of hematological response

    Time frame: After last consolidation cycle which is between 3 and 8 months

  4. Standardized evaluation of hematological response

    Time frame: Before HSCT

  5. Standardized evaluation of hematological response

    Time frame: at day 100 after HSCT

  6. Standardized evaluation of hematological response

    Time frame: at 5 years

  7. 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

  8. Minimal measurable residual disease (MRD) response

    Time frame: After first consolidation cycle which is between 1 and 2 months

  9. Minimal measurable residual disease (MRD) response

    Time frame: After last consolidation cycle which is between 3 and 8 months

  10. Minimal measurable residual disease (MRD) response

    Time frame: Before HSCT

  11. Minimal measurable residual disease (MRD) response

    Time frame: at day 100 after HSCT

  12. Minimal measurable residual disease (MRD) response

    Time frame: at 5 years

  13. Incidence of allogeneic HSCT

    Time frame: at 5 years

  14. Modalities of allogeneic HSCT

    Time frame: at 5 years

  15. Incidence of hematological relapses

    Time frame: at 5 years

  16. Type of hematological relapses

    Time frame: at 5 years

  17. Incidence of hematological progressions

    Time frame: at 5 years

  18. Type of hematological progressions

    Time frame: at 5 years

  19. Incidence of MRD relapses

    Time frame: at 5 years

  20. Incidence of MRD progressions

    Time frame: at 5 years

  21. Proportions of patients with treatment-related toxicities

    Time frame: at 5 years

    Treatment-related toxicities will be Evaluated by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

  22. Cumulative incidences of relapse

    Time frame: at 5 years

  23. Cumulative incidences of non-relapse mortality

    Time frame: at 5 years

  24. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: at inclusion

    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.

  25. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: 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 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.

  26. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: after 2 consolidations courses which is between 3 months and 8 months

    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.

  27. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: at 3 months after the end of treatment

    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.

  28. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: at 6 months after the end of treatment

    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.

  29. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: at 12 months after the end of treatment

    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.

  30. Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire

    Time frame: 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.

  31. Incidence of secondary cancer

    Time frame: at 5 years

  32. Incidence of secondary cancer

    Time frame: up to 15 years

Study contacts

Contact information is provided by the study sponsor or research team.

Hervé DOMBRET, Pr

CONTACT

[email protected]

1 57 27 68 47 ext. +33

Jérôme Lambert, Pr

CONTACT

[email protected]

142499742 ext. +33

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Acronym: eTHEMA

Important dates

Study start
2022
Primary completion
2042
Study completion
2042
First posted
Apr 14, 2022
Registry last updated
May 29, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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