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ctt-journal > Tretyakova et al. (Abstract)

Tretyakova et al. (Abstract)

Cellular Therapy and Transplantation (CTT), Vol. 3, No. 12
doi: 10.3205/ctt-2011-No12-abstract55

© The Authors. This abstract is provided under the following license: Creative Commons Attribution 3.0 Unported

Abstract accepted for "5th Raisa Gorbacheva Memorial Meeting Hematopoietic Stem Cell Transplantation in Children and Adults", Saint Petersburg, Russia, September 18–20, 2011

Preliminary Program

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Allogeneic hematopoietic stem cell transplantation in children and adolescents with myelodysplastic syndrome: a single center experience

Marya M. Tretyakova, Natalia V. Stancheva, Sergey N. Bondarenko, Olga A. Burdanova, Anastasya S. Borovkova, Svetlana V. Razumova, Tatiana A. Bykova, Andrey V. Kozlov, Elena V. Semenova, Ludmila S. Zubarovskaya, Boris V. Afanasyev

R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, St. Petersburg, Russia

Correspondence: Marya M. Tretyakova, R.M. Gorbacheva Memorial Institute of Children Hematology and Transplantation, St. Petersburg Pavlov State Medical University, 6/8, Tolstoy str., St. Petersburg, 197022, Russia, E-mail: tremmih@spam is badmail.ru

Abstract

Background: Myelodysplastic syndrome (MDS) is a very rare hematological malignancy in childhood, and its prognosis is very poor without allogeneic hematopoietic stem cell transplantation (allo-HSCT). Allo-HSCT is the only potentially curative treatment for MDS in children and young adults.

The aim of this study: To evaluate the efficacy of allo-HSCT in children and adolescents with MDS.

Patients and Methods: Allo-HSCT was performed in 21 patients (pts) with MDS including refractory anemia (n=1), refractory anemia with ringed sideroblasts (n=2), refractory anemia with excess of blasts (n=8), refractory cytopenia with multilineage dysplasia (n=1), juvenile myelomonocytic leukemia (n=1), and acute myeloid leukemia (AML) secondary to MDS (n=8). Six pts had monosomy 7. HLA-matched sibling HSCT (n=2), matched unrelated HSCT (n=13), and alternative HSCT (haploidentical (n=5), cord blood (n=1)) were performed. Bone marrow (n=9), peripheral blood stem cells (PBSC) (n=8), bone marrow and PBSC (n=4) were used as source of hematopoietic stem cells. The median age at transplant was 9.6 years (range 1–19). Male/female ratio was almost 1:1 (11/10). Fourteen and 7 pts received reduced intensity conditioning regimen (RIC) and myeloablative conditioning (MAC) respectively. MAC consisted of Busulfan (Bu) 16 mg/kg + Cyclophosphamide 120 mg/kg. RIC included Fludarabine (Flu) 150 mg/m2 + Melphalan (Mel) 140 mg/m2 in 3 pts, Flu 150–180 mg/m2 + Bu 8 mg/kg in 10 pts, and Flu 150 mg/m2 + Mel 140 mg/m2+ Thiotepa 10 mg/kg in 1 pt.

Results: The median time to neutrophil engraftment was 17 days (range 12–32). Primary engraftment failure occurred in 7 (33%) pts with RAEB and sAML. The estimated 1-year overall survival (OS) was 43%. There was a marked trend to better 1-year OS after bone marrow HSCT compared with peripheral blood HSCT (77% vs. 25%, p=ns) and MAC vs. RIC allo-HSCT (50% vs. 40%, p=ns). Patients with monosomy 7 had a 1-year OS of 14% whereas in patients with other cytogenetic abnormalities or with normal karyotype reached 57% (p<0.05).

Conclusions: Allo-HSCT is an effective treatment option for MDS in children and adolescents. Preliminary data indicate that using bone marrow graft and MAC is associated with better results. Monosomy 7 predicts an unfavorable prognosis.

Keywords: allo-HSCT, MDS, monosomy 7, myeloablative conditioning (MAC), reduced intensity conditioning regimen (RIC)