Renaud, Juliette; Goemans, Bianca F; Locatelli, Franco; Pigazzi, Martina; Redmond, Shelagh; Kuehni, Claudia E; Destaillats, Alice; Alonzo, Todd A; Gerbing, Robert B; Gamis, Alan; Aplenc, Richard; Renella, Raffaele; Cooper, Todd; Ceppi, Francesco (2024). Characteristics and treatment of acute myeloid neoplasms with cutaneous involvement in infants up to 6 months of age: A retrospective study. Pediatric blood & cancer, 71(7), e31006. Wiley 10.1002/pbc.31006
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BACKGROUND
Myeloid neoplasms account for 50% of cases of pediatric leukemias in infants. Approximately 25%-50% of patients with newborn leukemia have cutaneous extramedullary disease (EMD). In less than 10% of patients, aleukemic leukemia cutis or isolated extramedullary disease with cutaneous involvement (cEMD) occurs when skin lesions appear prior to bone marrow involvement and systemic symptoms. Interestingly, in acute myeloid leukemia with cutaneous EMD (AML-cEMD) and cEMD, spontaneous remissions have been reported.
METHOD
This is a multicentric retrospective cohort study aiming to describe characteristics, treatment, and outcome of infants with either cEMD or presence of cutaneous disease with involvement of the bone marrow (AML-cEMD). This study included patients born between 1990 and 2018 from Italy, the Netherlands, Switzerland, and the United States, diagnosed between 0 and 6 months of life with cEMD or AML-cEMD. Descriptive statistics, Fisher's exact test, Kaplan-Meier method, and log rank test were applied.
RESULTS
The cohort consisted of n = 50 patients, including 42 AML-cEMD and eight cEMD patients. The most common genetic mutation found was a KMT2A rearrangement (n = 26, 52%). Overall 5-year event-free survival (EFS) and overall survival (OS) were 66% [confidence interval (CI): 51-78] and 75% [CI: 60-85], respectively. In two patients, complete spontaneous remission occurred without any therapy. Central nervous system (CNS) involvement was found in 25% of cEMD patients. No difference in outcomes was observed between the AML-cEMD and cEMD groups, but none of the latter patients included in the study died. KMT2A rearrangements were not associated with poorer prognosis.
CONCLUSION
In the largest cohort to date, our study describes the characteristics of infants with cutaneous involvement of myeloid neoplasms including cytomolecular findings and survival rates. Further prospective biologic and clinical studies of these infants with myeloid neoplasms will be required to individualize therapy for this rare patient population.
Item Type: |
Journal Article (Original Article) |
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Division/Institute: |
04 Faculty of Medicine > Department of Gynaecology, Paediatrics and Endocrinology (DFKE) > Clinic of Paediatric Medicine 04 Faculty of Medicine > Pre-clinic Human Medicine > Institute of Social and Preventive Medicine (ISPM) |
UniBE Contributor: |
Redmond, Shelagh, Kühni, Claudia |
Subjects: |
600 Technology > 610 Medicine & health 300 Social sciences, sociology & anthropology > 360 Social problems & social services |
ISSN: |
1545-5009 |
Publisher: |
Wiley |
Language: |
English |
Submitter: |
Pubmed Import |
Date Deposited: |
15 Apr 2024 16:20 |
Last Modified: |
05 Jun 2024 17:39 |
Publisher DOI: |
10.1002/pbc.31006 |
PubMed ID: |
38616361 |
Additional Information: |
Open Access funding provided by Université de Lausanne. |
Uncontrolled Keywords: |
acute myeloid leukemia aleukemic leukemia cutis cutaneous infiltration extramedullary disease infants |
BORIS DOI: |
10.48350/195984 |
URI: |
https://boris.unibe.ch/id/eprint/195984 |
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