A Retrospective Epidemiologic Registry to Gain Insight Into the Characteristics and Prognosis of AML Patients According to the Routinely Used Genetic and Biologic Markers

NCT05541224 | Completed

• 1 other identifier: REAL-MOL
• Study Type: observational
• Target: 6,917
• Locations: 1 country
• Sites: 22

Brief Summary

This is a retrospective, translational, epidemiologic, multicenter, non-interventional study (No EPA study) to provide insights into disease epidemiology, disease biology, treatment regimens, and clinical outcomes of patients with acute myeloblastic leukemia (AML) in routine clinical practice according to their molecular markers. The primary objective of the study is to describe the use of the main molecular markers (FLT3 and NPM1) in the real-life according of the type of AML, treating institution, patients' characteristics, and disease status.

Conditions

Acute Myeloid Leukemia

Keywords

De novo Acute Myeloid Leukemia; Relapsed/refractory Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Percentage of patients with molecular alterations (FLT3 and NPM1 mutations).

  Percentage of each of the standard screening panel molecular alterations studied in the AML patients (FLT3 and NPM1) by AML type, by center, by patient's characteristics and the disease status.

  Baseline

Secondary Outcomes (4)

• Overall response rate.

  Throughout the study period. Approximately 1 year

• Description of initial and later treatments in each baseline alterations studied (FLT3 and NPM1) in each AML type.

  Baseline and throughout the study period. Approximately 1 year

• Molecular response rate.

  Throughout the study period. Approximately 1 year

• Description of biology of disease at relapse or refractoriness by molecular characteristics.

  Baseline and throughout the study period. Approximately 1 year

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Adult patients diagnosed with AML (new diagnosis or relapsed/refractory disease). These patients must meet all the inclusion criteria and none of the exclusion criteria specified.

You may qualify if:

• All adult patients with AML (excluding APL) according to the WHO criteria (2008), regardless of the treatment administered by their treating physician.
• AML at diagnosis and at relapse or refractoriness.
• Patients from institutions participating in the ongoing PETHEMA AML registry.
• Patients with information about the molecular screening including FLT3 with or without NPM1 mutations (including positive/negative or not performed).
• Ability to give informed consent before the study initiation. Death patients and patients who are no longer contactable or lost to follow-up will be excluded from consent requirement.

You may not qualify if:

• Pediatric patients.
• Acute promyelocytic leukemia.

Sponsors & Collaborators

• PETHEMA Foundation: lead
• Novartis: collaborator

Study Sites (22)

Hospital General Universitario de Albacete

Albacete, Spain

Location

Hospital General Universitario de Alicante

Alicante, Spain

Location

Hospital Germans Trias i Pujol-ICO

Badalona, Spain

Location

Hospital Universitario de Burgos

Burgos, Spain

Location

Hospital General Universitario de Castellón

Castellon, Spain

Location

Hospital San Pedro de Alcántara

Cáceres, Spain

Location

Hospital Universitario Reina Sofía

Córdoba, Spain

Location

Hospital Universitario de Gran Canaria Doctor Negrín

Las Palmas de Gran Canaria, Spain

Location

Hospital Universitario de León

León, Spain

Location

Hospital Universitario Lucus Augusti

Lugo, Spain

Location

Hospital Clínico San Carlos

Madrid, Spain

Location

Hospital Univeristario Ramón y Cajal

Madrid, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, Spain

Location

Hospital Universitario Fundación Jiménez Díaz

Madrid, Spain

Location

Hospital General Universitario Morales Meseguer

Murcia, Spain

Location

Hospital Universitario Central Asturias

Oviedo, Spain

Location

Clínica Universitaria de Navarra

Pamplona, Spain

Location

Hospital Universitario de Salamanca

Salamanca, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, Spain

Location

Hospital Clínico Universitario de Valencia

Valencia, Spain

Location

Hospital Universitari I Politécnic La Fe

Valencia, Spain

Location

Hospital Universitario Doctor Peset

Valencia, Spain

Location

Related Publications (13)

• Schlenk RF, Dohner K, Krauter J, Frohling S, Corbacioglu A, Bullinger L, Habdank M, Spath D, Morgan M, Benner A, Schlegelberger B, Heil G, Ganser A, Dohner H; German-Austrian Acute Myeloid Leukemia Study Group. Mutations and treatment outcome in cytogenetically normal acute myeloid leukemia. N Engl J Med. 2008 May 1;358(18):1909-18. doi: 10.1056/NEJMoa074306.

  PMID: 18450602 BACKGROUND

• Thiede C, Steudel C, Mohr B, Schaich M, Schakel U, Platzbecker U, Wermke M, Bornhauser M, Ritter M, Neubauer A, Ehninger G, Illmer T. Analysis of FLT3-activating mutations in 979 patients with acute myelogenous leukemia: association with FAB subtypes and identification of subgroups with poor prognosis. Blood. 2002 Jun 15;99(12):4326-35. doi: 10.1182/blood.v99.12.4326.

  PMID: 12036858 BACKGROUND

• Frohling S, Schlenk RF, Breitruck J, Benner A, Kreitmeier S, Tobis K, Dohner H, Dohner K; AML Study Group Ulm. Acute myeloid leukemia. Prognostic significance of activating FLT3 mutations in younger adults (16 to 60 years) with acute myeloid leukemia and normal cytogenetics: a study of the AML Study Group Ulm. Blood. 2002 Dec 15;100(13):4372-80. doi: 10.1182/blood-2002-05-1440. Epub 2002 Aug 8.

  PMID: 12393388 BACKGROUND

• Kottaridis PD, Gale RE, Frew ME, Harrison G, Langabeer SE, Belton AA, Walker H, Wheatley K, Bowen DT, Burnett AK, Goldstone AH, Linch DC. The presence of a FLT3 internal tandem duplication in patients with acute myeloid leukemia (AML) adds important prognostic information to cytogenetic risk group and response to the first cycle of chemotherapy: analysis of 854 patients from the United Kingdom Medical Research Council AML 10 and 12 trials. Blood. 2001 Sep 15;98(6):1752-9. doi: 10.1182/blood.v98.6.1752.

  PMID: 11535508 BACKGROUND

• Gale RE, Hills R, Kottaridis PD, Srirangan S, Wheatley K, Burnett AK, Linch DC. No evidence that FLT3 status should be considered as an indicator for transplantation in acute myeloid leukemia (AML): an analysis of 1135 patients, excluding acute promyelocytic leukemia, from the UK MRC AML10 and 12 trials. Blood. 2005 Nov 15;106(10):3658-65. doi: 10.1182/blood-2005-03-1323. Epub 2005 Aug 2.

  PMID: 16076872 BACKGROUND

• Rombouts WJ, Blokland I, Lowenberg B, Ploemacher RE. Biological characteristics and prognosis of adult acute myeloid leukemia with internal tandem duplications in the Flt3 gene. Leukemia. 2000 Apr;14(4):675-83. doi: 10.1038/sj.leu.2401731.

  PMID: 10764154 BACKGROUND

• Yokota S, Kiyoi H, Nakao M, Iwai T, Misawa S, Okuda T, Sonoda Y, Abe T, Kahsima K, Matsuo Y, Naoe T. Internal tandem duplication of the FLT3 gene is preferentially seen in acute myeloid leukemia and myelodysplastic syndrome among various hematological malignancies. A study on a large series of patients and cell lines. Leukemia. 1997 Oct;11(10):1605-9. doi: 10.1038/sj.leu.2400812.

  PMID: 9324277 BACKGROUND

• Whitman SP, Archer KJ, Feng L, Baldus C, Becknell B, Carlson BD, Carroll AJ, Mrozek K, Vardiman JW, George SL, Kolitz JE, Larson RA, Bloomfield CD, Caligiuri MA. Absence of the wild-type allele predicts poor prognosis in adult de novo acute myeloid leukemia with normal cytogenetics and the internal tandem duplication of FLT3: a cancer and leukemia group B study. Cancer Res. 2001 Oct 1;61(19):7233-9.

  PMID: 11585760 BACKGROUND

• Dohner K, Schlenk RF, Habdank M, Scholl C, Rucker FG, Corbacioglu A, Bullinger L, Frohling S, Dohner H. Mutant nucleophosmin (NPM1) predicts favorable prognosis in younger adults with acute myeloid leukemia and normal cytogenetics: interaction with other gene mutations. Blood. 2005 Dec 1;106(12):3740-6. doi: 10.1182/blood-2005-05-2164. Epub 2005 Jul 28.

  PMID: 16051734 BACKGROUND

• Verhaak RG, Goudswaard CS, van Putten W, Bijl MA, Sanders MA, Hugens W, Uitterlinden AG, Erpelinck CA, Delwel R, Lowenberg B, Valk PJ. Mutations in nucleophosmin (NPM1) in acute myeloid leukemia (AML): association with other gene abnormalities and previously established gene expression signatures and their favorable prognostic significance. Blood. 2005 Dec 1;106(12):3747-54. doi: 10.1182/blood-2005-05-2168. Epub 2005 Aug 18.

  PMID: 16109776 BACKGROUND

• Suzuki T, Kiyoi H, Ozeki K, Tomita A, Yamaji S, Suzuki R, Kodera Y, Miyawaki S, Asou N, Kuriyama K, Yagasaki F, Shimazaki C, Akiyama H, Nishimura M, Motoji T, Shinagawa K, Takeshita A, Ueda R, Kinoshita T, Emi N, Naoe T. Clinical characteristics and prognostic implications of NPM1 mutations in acute myeloid leukemia. Blood. 2005 Oct 15;106(8):2854-61. doi: 10.1182/blood-2005-04-1733. Epub 2005 Jun 30.

  PMID: 15994285 BACKGROUND

• Dohner H, Estey E, Grimwade D, Amadori S, Appelbaum FR, Buchner T, Dombret H, Ebert BL, Fenaux P, Larson RA, Levine RL, Lo-Coco F, Naoe T, Niederwieser D, Ossenkoppele GJ, Sanz M, Sierra J, Tallman MS, Tien HF, Wei AH, Lowenberg B, Bloomfield CD. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017 Jan 26;129(4):424-447. doi: 10.1182/blood-2016-08-733196. Epub 2016 Nov 28.

  PMID: 27895058 BACKGROUND

• Hellmann F, Verdi M, Schlemper BR Jr, Caponi S. 50th anniversary of the Declaration of Helsinki: the double standard was introduced. Arch Med Res. 2014 Oct;45(7):600-1. doi: 10.1016/j.arcmed.2014.10.005. Epub 2014 Oct 31.

  PMID: 25450586 BACKGROUND

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Pau Montesinos Fernández

  Hospital Universitari i Politècnic La Fe (Valencia)

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2022
• First Posted: September 15, 2022
• Study Start: November 1, 2019
• Primary Completion: October 1, 2020
• Study Completion: October 1, 2020
• Last Updated: September 15, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (22)