Diagnosis and treatment of juvenile myelomonocytic leukemia

ABSTRACT

Objective: To investigate clinical features, diagnosis, treatment strategies and prognosis of juvenile myelomonocytic leukemia (JMML).

Methods: The clinical data of 21 patients with JMML who were diagnosed in our hospital from January 2013 to May 2018 were retrospectively analyzed.

Results: Among the 21 children with JMML, 16 were male and 5 were female. Out of the 21 children who were diagnosed with JMML, 7 were lost after treatment while the remaining 14 received A-3V chemotherapy regimen of South Korea. The effective response rate was 78.5%. The three-year overall survival (OS) rate and three-year disease-free survival (DFS) rate were (76.2 ± 14.8)% and (66.2 ± 14)%, respectively. Single factor analysis showed that PLT count ≤33×109/L, LDH level >500 U/L and HbF level >10% and chemotherapy only were the significant factors that lead to poor prognosis in children. Cox multivariate analysis showed that the choice of treatment options affected the prognosis of JMML children. By taking prognostic factors for long-term efficacy into account, patients with treatment strategy of chemotherapy alongside hematopoietic stem cell transplantation (HSCT) have a better prognosis.

Conclusion: The PLT count, LDH level, HbF level and choice of treatment plan are important for the evaluation of prognosis for children with JMML. Although there is a lack of consistency in terms of donors but the A-3V scheme is relatively stable, so HSCT should be preferred for children with poor prognostic factors.

KEYWORDS:

• Juvenile myelomonocytic leukemia
• clinical features
• diagnosis
• A-3V scheme
• treatment
• prognosis
• hematopoietic stem cell transplantation
• overall survival
• disease-free survival

Acknowledgements

The study has been approved by Sun Yat-sen Memorial Hospital Ethics Committee (China). Authors gratefully acknowledge the help of all parents and children.

Availability of data and materials

The data sets used and/or analyzed during the current study are not available because of privacy.

Disclosure statement

No potential conflict of interest was reported by the authors.

ORCID

Kun-yin Qiu http://orcid.org/0000-0002-2865-4685

Additional information

Funding

This work was supported by the National Natural Science Foundation of China [grant number 81570140].