Spontaneous remission and loss of monosomy 7: a window of opportunity for young children with SAMD9L syndrome

Haematologica. 2024 Feb 1;109(2):422-430. doi: 10.3324/haematol.2023.283591.

Abstract

Monosomy 7 is the most common cytogenetic abnormality in pediatric myelodysplastic syndrome (MDS) and associated with a high risk of disease progression. However, in young children, spontaneous loss of monosomy 7 with concomitant hematologic recovery has been described, especially in the presence of germline mutations in SAMD9 and SAMD9L genes. Here, we report on our experience of close surveillance instead of upfront hematopoietic stem cell transplantation (HSCT) in seven patients diagnosed with SAMD9L syndrome and monosomy 7 at a median age of 0.6 years (range, 0.4-2.9). Within 14 months from diagnosis, three children experienced spontaneous hematological remission accompanied by a decrease in monosomy 7 clone size. Subclones with somatic SAMD9L mutations in cis were identified in five patients, three of whom attained hematological remission. Two patients acquired RUNX1 and EZH2 mutations during the observation period, of whom one progressed to myelodysplastic syndrome with excess of blasts (MDS-EB). Four patients underwent allogeneic HSCT at a median time of 26 months (range, 14-40) from diagnosis for MDSEB, necrotizing granulomatous lymphadenitis, persistent monosomy 7, and severe neutropenia. At last follow-up, six patients were alive, while one passed away due to transplant-related causes. These data confirm previous observations that monosomy 7 can be transient in young children with SAMD9L syndrome. However, they also indicate that delaying HSCT poses a substantial risk of severe infection and disease progression. Finally, surveillance of patients with SAMD9L syndrome and monosomy 7 is critical to define the evolving genetic landscape and to determine the appropriate timing of HSCT (clinicaltrials gov. Identifier: NCT00662090).

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Child
  • Child, Preschool
  • Chromosome Deletion*
  • Chromosomes, Human, Pair 7 / genetics
  • Disease Progression
  • Humans
  • Infant
  • Intracellular Signaling Peptides and Proteins / genetics
  • Monosomy
  • Myelodysplastic Syndromes* / diagnosis
  • Myelodysplastic Syndromes* / genetics
  • Myelodysplastic Syndromes* / therapy
  • Remission, Spontaneous
  • Transcription Factors / genetics

Substances

  • Transcription Factors
  • SAMD9 protein, human
  • Intracellular Signaling Peptides and Proteins

Supplementary concepts

  • Chromosome 7, monosomy

Associated data

  • ClinicalTrials.gov/NCT00662090

Grants and funding

Funding: This work was generated within the European Reference Network for Pediatric Cancer (PAEDCAN). It was supported by the German Federal Ministry of Education and Research (BMBF) 01GM1911A “MyPred - Network for young individuals with syndromes predisposing to myeloid malignancies” to BS, CMN, GG, ME, AY and MWW.