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

Korthof, Elisabeth T.; Snijder, P P; Graaff, Aisha A. de; Lankester, Arjan C.; Bredius, Robbert G. M.; Ball, Lynne M.; Lie, J.L.W.T.; Vossen, Jaak M.; Egeler, R. Maarten

doi:10.1038/sj.bmt.1704778

Cited by 19 publications

(15 citation statements)

References 23 publications

(37 reference statements)

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“…23 However, in an evaluation of 21 patients with mixed chimerism after initial HSCT for JMML, only 1 patient was alive and in remission for >1500 days after receiving DLI. 24 Moreover, all 21 patients in the study with mixed chimerism experienced a hematological relapse, further clarifying mixed chimerism status as a risk factor.…”

Section: Discussionmentioning

confidence: 99%

Cytosine Arabinoside and Mitoxantrone Followed by Second Allogeneic Transplant for the Treatment of Children With Refractory Juvenile Myelomonocytic Leukemia

Patel

Coulter

Grovas

et al. 2014

Journal of Pediatric Hematology/Oncology

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Summary Hematopoietic stem cell transplantation (HSCT) remains the only curative option for most patients with juvenile myelomonocytic leukemia (JMML). However, persistent disease and relapse rates after transplant range from 26% to 58%. We report the successful use of second HSCT after preparation with mitoxantrone and cytosine arabinoside (Ara-C) for patients with refractory or recurrent disease. Between 1993 and 2006, 5 children who underwent HSCT at our institution as initial therapy for JMML had persistent disease or relapsed. Pre-HSCT conditioning varied and donors were either HLA-matched siblings (n = 2) or matched unrelated donors (n = 3). After initial HSCT, they subsequently received high-dose Ara-C (3 g/m2 IV) every 12 hours on days −8 through −3 and mitoxantrone (10 mg/m2/d IV) on days − 8, − 7, − 6 followed by second HSCT from their original donors. All 5 patients are alive at 88, 179, 199, 234, and 246 months with no evidence of JMML, no significant toxicity, and 100% donor chimera as determined by PCR short-tandem repeat analysis. Our experience supports second transplant utilizing high-dose Ara-C and mitoxantrone in children with JMML who do not respond or relapse after first transplant.

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Section: Discussionmentioning

confidence: 99%

Cytosine Arabinoside and Mitoxantrone Followed by Second Allogeneic Transplant for the Treatment of Children With Refractory Juvenile Myelomonocytic Leukemia

Patel

Coulter

Grovas

et al. 2014

Journal of Pediatric Hematology/Oncology

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“…It ought to be beneficial for JMML patients undergoing HSCT. We thank Koyama et al for their comments on our paper 1 and on another recently published patient series, 2 and for adding their own interesting case. Since the relapse incidence in hematopoietic stem cell transplantation (HSCT) for juvenile myelomonocytic leukemia (JMML) is around 35% in almost every recent series, 1 it is important to keep the discussion alive to continue improving the treatment of children with JMML.…”

Section: -10mentioning

confidence: 99%

“…1 Allogeneic hematopoietic stem cell transplantation (HSCT) is presently the only curative treatment for JMML, because the disease usually progresses rapidly. Leukemia recurrence represents the main cause of treatment failure in children with JMML given HSCT, with the relapse rate being as high as 50%.…”

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confidence: 99%

Successful treatment of JMML with related bone marrow transplantation after reduced-intensity conditioning

Koyama

Nakano

Takeshita

et al. 2005

Bone Marrow Transplant

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“…With respect to risk for graft failure, patients with BM failure as original disease, that is, children with severe aplastic anaemia and Fanconi anaemia (n ¼ 15), were further categorized as either at increased risk when X20 erythrocyte or thrombocyte transfusions had been given in the preconditioning period or at standard risk. With respect to risk for relapse of the haematological malignancy after BMT, patients transplanted for such a diagnosis (n ¼ 78) were further categorized as at increased risk, that is, myelodysplastic syndromes in transformation or blast crisis, [34][35][36] juvenile myelomonocytic leukemia, 37,38 Ph þ CML longer than 2 years in the first chronic phase or in advanced phase, 39,40 secondary AML in first complete remission, AML in the second complete remission, 41 ALL in the second complete remission after early BM relapse 42 and ALL in the third complete remission, vs at standard risk (n ¼ 40 vs 38, see Table 1). …”

Section: Variables Evaluatedmentioning

confidence: 99%

Unrelated donor marrow transplantation in children: transplant policy and outcome in Leiden Paediatrics SCT-Centre

Vossen

Donker

Heemskerk

et al. 2009

Bone Marrow Transplant

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The transplant policy for unrelated donor (UD) BMT at Leiden Paediatrics' SCT-Centre consisted of the use of (1) fully HLA-matched donors or, if not available, HLA-class I matched and/or cytotoxic T-lymphocyte precursor (CTLp)-negative donors and (2) protective isolation of the recipient and antimicrobial suppression of his/her gut microflora to prevent infections and acute GVHD. Engraftment, GVHD, relapse in the case of malignancy and survival were studied retrospectively in 126 evaluable children, transplanted between 1988 and 2005. In addition to the effect of HLAmatching, that of other transplant-relevant variables on the outcome was also studied. Actuarial OS was 65% and the EFS was 59%, 13% graft failures occurred and 7.5% Xgrade II acute GVHD. HLA-class II mismatches combined with HLA-class I matches resulted in a superior OS of 92%, as did a negative vs positive CTLp test, that is, 65 vs 33%. Analysis of other variables showed a poorer OS in patients X10 yrs vs o10 yrs, that is, 54 vs 73%, and in male recipients of a female donor graft, that is, 53 vs 69% for other combinations. UD-BMT can be optimized by permitting HLA-class I-matched and/or CTLp-negative donors, and probably by choosing male donors for male recipients.

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

Cited by 19 publications

References 23 publications

Cytosine Arabinoside and Mitoxantrone Followed by Second Allogeneic Transplant for the Treatment of Children With Refractory Juvenile Myelomonocytic Leukemia

Cytosine Arabinoside and Mitoxantrone Followed by Second Allogeneic Transplant for the Treatment of Children With Refractory Juvenile Myelomonocytic Leukemia

Successful treatment of JMML with related bone marrow transplantation after reduced-intensity conditioning

Unrelated donor marrow transplantation in children: transplant policy and outcome in Leiden Paediatrics SCT-Centre

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