Session: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster I
Hematology Disease Topics & Pathways:
MDS, Bone Marrow Failure Syndromes, Chronic Myeloid Malignancies, Pediatric, Diseases, Myeloid Malignancies, Study Population, Human
Method: We analyzed 101 consecutive pediatric patients (< 18 years) diagnosed with RCC, normal karyotype and absence of genetic predisposition who did neither receive HSCT nor immunosuppressive therapy within 2 years from diagnosis. All patients were enrolled in the German EWOG-MDS registries between 1998 and 2021 with informed consent from parental guardians. The research was approved by the University of Freiburg institutional ethics committee.
Results: The median age at diagnosis of the 55 males and 46 females was 10.1 years (1.4-17.3). The median white blood count was 3.4 G/L, and 12% and 30 % of patients had an absolute neutrophil count < 0.5 G/L and ≥ 1.5 G/L, respectively. Anemia was frequent and the platelet count was < 100 G/L in 74% of patients. The majority of patients (84%) had a hypocellular bone marrow. Patients were followed up till adulthood with a median follow up time of 7 years. Clonal evolution defined by an abnormal karyotype occurred in 3 patients, one of them had disease progression to MDS with excess blasts. In another 3 patients (3%), clinical paroxysmal nocturnal hemolysis (PNH) developed. Nine patients (9%) received allogeneic hematopoietic stem cell transplantation (HSCT) during the clinical course for clonal evolution (n=1), PNH (n=2) or cytopenia (n=6). All patients were alive at last follow up. Five and 10 years HSCT-free survival was 94% and 83%, respectively. Most of the patients had persistent cytopenia and/or elevated MCV over the course of disease. At last follow-up, in 15 patients without HSCT (16%) normal blood counts and a normal MCV was documented.
Conclusions: Our results indicate that in the absence of a known predisposition syndrome, pediatric patients with RCC and a normal karyotype can safely be managed by a watch and wait strategy unless transfusion dependency or severe neutropenia require HSCT. The persistence of cytopenia during the disease course in the vast majority of patients suggests that the RCC histopathology is the morphological phenotype of an intrinsic bone marrow defect. Furthermore, it emphasizes the need for a structured transition from pediatric to adult hematology to offer the affected patients long-term clinical surveillance.
Disclosures: Drexler: Novartis: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding, Speakers Bureau; Sobi: Membership on an entity's Board of Directors or advisory committees, Research Funding; Alexion: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding. Wlodarski: OLG Research & Consulting: Consultancy; Guidepoint: Consultancy. Beier: Pfizer: Honoraria; Alexion: Honoraria; Sobi: Honoraria; RepeatDx: Other: Scientifc collaboration. Strahm: Pfizer: Membership on an entity's Board of Directors or advisory committees. Niemeyer: Novartis: Consultancy; BMS: Membership on an entity's Board of Directors or advisory committees.
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