Clinical trial NCT05326919: The Patient Cohort of the National Center for Precision Medicine in Leukemia
Official title: The Patient Cohort of the National Center for Precision Medicine in Leukemia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Acute Myeloid Leukemia
Acute Lymphoblastic Leukemia
High-risk Myelodysplastic Syndrome
Secondary Myelofibrosis in Myeloproliferative Disease
Myeloproliferative Neoplasm, Unclassifiable
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2022-04-14
Enrollment: 3000
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

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.


Eligibility criteria: 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

Primary outcomes:
measure: Event Free Survival
timeFrame: at 5 years
measure: Relapse Free Survival
timeFrame: at 5 years
measure: Overall Survival
timeFrame: at 5 years
Secondary outcomes:
measure: Standardized evaluation of hematological response
timeFrame: 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
measure: Standardized evaluation of hematological response
timeFrame: After first consolidation cycle which is between 1 and 2 months
measure: Standardized evaluation of hematological response
timeFrame: After last consolidation cycle which is between 3 and 8 months
measure: Standardized evaluation of hematological response
timeFrame: Before HSCT
measure: Standardized evaluation of hematological response
timeFrame: at day 100 after HSCT
measure: Standardized evaluation of hematological response
timeFrame: at 5 years
measure: Minimal measurable residual disease (MRD) response
timeFrame: 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
measure: Minimal measurable residual disease (MRD) response
timeFrame: After first consolidation cycle which is between 1 and 2 months
measure: Minimal measurable residual disease (MRD) response
timeFrame: After last consolidation cycle which is between 3 and 8 months
measure: Minimal measurable residual disease (MRD) response
timeFrame: Before HSCT
measure: Minimal measurable residual disease (MRD) response
timeFrame: at day 100 after HSCT
measure: Minimal measurable residual disease (MRD) response
timeFrame: at 5 years
measure: Incidence of allogeneic HSCT
timeFrame: at 5 years
measure: Modalities of allogeneic HSCT
timeFrame: at 5 years
measure: Incidence of hematological relapses
timeFrame: at 5 years
measure: Type of hematological relapses
timeFrame: at 5 years
measure: Incidence of hematological progressions
timeFrame: at 5 years
measure: Type of hematological progressions
timeFrame: at 5 years
measure: Incidence of MRD relapses
timeFrame: at 5 years
measure: Incidence of MRD progressions
timeFrame: at 5 years
measure: Proportions of patients with treatment-related toxicities
timeFrame: at 5 years
measure: Cumulative incidences of relapse
timeFrame: at 5 years
measure: Cumulative incidences of non-relapse mortality
timeFrame: at 5 years
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: at inclusion
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: 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
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: after 2 consolidations courses which is between 3 months and 8 months
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: at 3 months after the end of treatment
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: at 6 months after the end of treatment
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: at 12 months after the end of treatment
measure: Quality-of-life assessed using the EORTC-QLQ-C30 v3 questionnaire
timeFrame: at day 100 after hematopoietic stem cell transplant
measure: Incidence of secondary cancer
timeFrame: at 5 years
measure: Incidence of secondary cancer
timeFrame: up to 15 years
Other outcomes:
Locations:
city: Bobigny
country: France
facility: Hôpital Avicenne
city: Paris
country: France
facility: Hopital Robert Debré
city: Paris
country: France
facility: Hôpital Saint Louis
Study officials:
References: