Allogeneic hematopoietic stem cell transplantation for 27 children with juvenile myelomonocytic leukemia diagnosed based on the criteria of the International JMML Working Group

Abstract

Prognostic factors of juvenile myelomonocytic leukemia (JMML) have not been clarified because of its very low incidence and inaccuracy in the diagnosis. The purpose of this study was to evaluate children with JMML given an allogeneic hematopoietic stem cell transplantation (SCT) and the role of different variables potentially influencing outcome in a nationwide survey in Japan based on the newly proposed criteria by the International JMML Working Group. The study patients were 27 children who underwent SCT among 55 JMML patients retrospectively collected in the survey. The source of grafts was HLA-identical siblings in 12 cases, HLA-matched unrelated individuals in 10 and others in five. Total body irradiation was used in 18 cases. Event-free and overall survival (OS) at 4 years after SCT were 54.2 ± 11.2% (s.e.) and 57.9 ± 11.0% (s.e.), respectively. Six patients died of relapse and three of complications. Patients with abnormal karyotypes showed a significantly lower OS than those with normal karyotypes (P Ͻ 0.001). Patients below 1 year of age showed a significantly higher OS than those of 1 year of age or more (P = 0.02). Patients with grade 0-1 acute graft-versus-host disease (GVHD) or chronic GVHD had a more favorable OS than those without them, although they were not statistically significant (P Ͼ 0.05). Other variables studied were not associated with OS. A multivariate analysis of these factors yielded the abnormal karyotype as the only significant risk factor for lower OS (risk ratio: 11.0; 95% CI: 2.7-45.1). Leukemia (2002) 16, 645-649.

Allogeneic bone marrow transplantation for juvenile myelomonocytic leukemia: a single center experience of 23 patients

“…8,10,11,14,15 In our series, OS is 43.5710% at 5 and 10 years; the probability of relapse-free survival (TRM censured) is 48712%. Rapid sustained engraftment in 20 of 22 evaluable children proved that engraftment was not a problem, similar to results in other studies.…”

“…9,10 Allogeneic BMT has been shown to improve outcome: retrospective evaluations of series including 15 patients or more reported an overall survival (OS) of 39-58%. 2,[11][12][13][14][15] Woods 10 found a similar OS at 6 years of 31% for 13 patients treated on a prospective study (CCG protocol 2891). The major causes of failure in all series were a high relapse rate and a high transplant-related mortality (TRM).…”

“…In retrospect, all patients qualified for the diagnosis of JMML based on fulfilment of the three major and at least two of the five minor criteria of the International JMML Working Group. 14 …”

Juvenile Myelomonocytic Leukemia in India: Cure Remains a Distant Dream!

“…Both patients who received chemotherapy died (one at our centre and second at another centre). The only curative therapy is allogeneic SCT which is able to cure *50 % of patients [5,6]. There are no reports of SCT for JMML from India and in our series three children successfully underwent allogeneic SCT.…”

“…Conventional chemotherapy rarely achieves cure [1]. However, survival up to 57-64 % have been reported post allogeneic stem cell transplant (SCT) [5,6]. There is a paucity of outcome data for JMML from India [7][8][9].…”