ISSN 1866-8836
Клеточная терапия и трансплантация

PO-01. Hematopoietic stem cell transplantation in rare pediatric cancers and hematological conditions: the N. N. Blokhin National Medical Research Center of Oncology experience

Teymur Z. Aliev, Irina O. Kostareva, Nara G. Stepanyan, Natalia A. Burlaka, Karina A. Sergeenko, Yuri V. Lozovan, Elena B. Machneva, Olga A. Kapkova, Amina M. Suleimanova, Tatiana L. Ushakova, Marina V. Rubanskaya, Natalia A. Batmanova, Timur T. Valiev, Anatoly P. Kazantsev, Vladimir G. Polyakov, Kirill I. Kirgizov, Svetlana R. Varfolomeeva

N. N. Blokhin National Medical Research Center of Oncology, Moscow, Russia

Contact: Dr. Teymur Z. Aliev, phone: +7 (916) 368-90-27, e-mail:

doi 10.18620/ctt-1866-8836-2022-11-3-1-132


Hematopoietic stem cell transplantation (HSCT) is one of the key stages in treatment of children with malignant and benign conditions. In some cases HSCT is the only radical treatment method possible. It is also used in rare malignant and non-malignant pediatric hematological diseases. In some cases these non-malignant diseases belong to the group of so-called precancerous states, which makes the topic relevant for specialists working with pediatric neoplasms. We present the N. N. Blokhin NMRCO experience of HSCT in children with rare oncological and hematological diseases.

Materials and methods

At the N. N. Blokhin NMRCO, a total of 17 pediatric patients (median age 47 months, range 25-192 months; M/F=8/9) with rare oncological and hematological diseases received HSCT for retinoblastoma (RB, n=3), germ cell tumor (GCT, n=8), Fanconi anemia (FA, n=2), dyskeratosis congenita (DC, n=3), and pleuropulmonary blastoma (PPB, n=1) in 2019-2022. The patients underwent autologous (auto-HSCT) and allogeneic (allo-HSCT) transplantations. All patients underwent pharmacological conditioning. In children with AF and DC (allo-HSCT) in included Busulfan, Fludarabine, ATG and Cyclophosphamide. In children with RB and GCT (auto-HSCT) it consisted of Etoposide, Thiotepa, Carboplatin and Cyclophosphamide, while in a patient with PPB (auto-HSCT) of Treosulfan and Melphalan. The HSC source was peripheral blood (PB) in auto-HSCT, and bone marrow (BM) from a sibling (n=4) or PSC from an unrelated donor (n=1) in allo-HSCT recipients.


All patients successfully underwent HSCT. In early post-HSCT stages the following complications were registered: Gr 1-2 skin toxicity, Gr 1-2 oropharyngeal mucositis, Gr 1-2 neutropenic enterocolitis. These complications were successfully controlled by standard therapeutic interventions. Signs of Gr 1 acute GVHD were observed in 2 allo-HSCT recipients. Average time to engraftment after auto-HSCT was D+12, and D+19 after allo-HSCT. No significant toxicity was recorded. One death was observed in a child with RB 7 months after auto-HSCT due to relapse. The median follow-up is currently 9 (1-25) months.


HSCT is an acceptable option in children with rare oncological/hematological conditions. Each patient with such condition requires an individual approach to management during HSCT and subsequent follow-up.


Hematopoietic stem cell transplantation, oncology, hematology, rare diseases, children.

Volume 11, Number 3

Download PDF version

doi 10.18620/ctt-1866-8836-2022-11-3-1-132

Back to the list