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

Recruiting

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

• 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

Study Timeline

• Study First Submitted: 2022-01-28
• Study Start: 2022-03-28
• Study First Submitted QC: 2022-04-07
• Study First Posted: 2022-04-14
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-26
• Last Update Posted: 2024-05-29
• Primary Completion: 2042-03
• Study Completion: 2042-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 3000

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Relapse Free Survival	at 5 years
Overall Survival	at 5 years
Event Free Survival	at 5 years

Secondary Outcome Measures

Name	Time	Method
Incidence of secondary cancer	up to 15 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
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.
Modalities of allogeneic HSCT	at 5 years
Incidence of MRD progressions	at 5 years
Cumulative incidences of non-relapse mortality	at 5 years

Trial Locations

Locations (3)

Hôpital Avicenne; 🇫🇷 Bobigny, France

Hopital Robert Debré; 🇫🇷 Paris, France

Hôpital Saint Louis; 🇫🇷 Paris, France