Our initial experience with hematopoietic stem cell transplantation (HSCT) from a matched unrelated donor (MUD; n = 12) or a haploidentical related donor (n = 6) with T cell receptor (TCR)αβ/CD19 graft depletion in patients with Wiskott-Aldrich syndrome (WAS) (n = 18) showed a dramatic decrease in the incidence of graft-versus-host disease (GVHD) and transplantation-related mortality, with an increased overall survival (OS) of 88.9%. Unfortunately, the treatment was associated with mixed myeloid donor chimerism and secondary graft dysfunction (severe thrombocytopenia, n = 2; graft rejection, n = 5). To improve the outcome, we hypothesized that the addition of G-CSF and plerixafor to the conditioning chemotherapy would result in more complete donor stem cell engraftment. This trial was registered at www.clinicaltrials.gov (NCT03019809). A study group of patients with WAS (n = 16) underwent TCRαβ/CD19-depleted HSCT (MUD, n = 6; haploidentical, n = 10). The conditioning regimen was treosulfan-fludarabine-rabbit antithymocyte globulin-melphalan (or thiophosphamide in 1 patient) with G-CSF (10 µg/kg/day for 5 days starting on day -8) and plerixafor (240 µg/kg/day for 3 days starting on day -6). The clinical outcomes in this study were compared to those in a historical dataset (n = 18). No patients had grade III/IV acute GVHD in either the study or the historical control group. Importantly, in the patients with WAS, there was no statistical significance in OS between those who underwent HSCT from haploidentical donors and those who underwent HSCT from MUDs (93.8% versus 88.5%; P = .612). All patients in the study group had full donor chimerism in whole blood and in the CD3 compartments. The OS was 93.8%, and there were no cases of graft dysfunction. This study demonstrates the efficacy of adding G-CSF/plerixafor to the conditioning regimen before HSCT with TCRαβ/C D19 graft depletion in patients with WAS.

Summary:Allogeneic hematopoietic stem cell transplantation (SCT) from unaffected donors remains the only modality for the correction of hematological abnormalities in Fanconi anemia (FA) patients. We performed four HLA-matched related donor SCT using a novel irradiation and cyclophosphamide-free conditioning regimen. The protocol included fludarabine 150 mg/m 2 , busulfan 4 mg/kg, and antithymocyte globulin 90 mg/kg. Graft-versus-host disease (GVHD) prophylaxis was cyclosporin A, MTX, and daclizumab. The engraftment and occurrence of full stable donor hemopoiesis was rapid in all cases with minimal short-term toxic complications. There were no infections or febrile episodes during the inpatient phase. Three patients developed acute GVHD grade I-II involving gut and skin and one patient progressed to extensive chronic GVHD. The preparative conditioning regimen is safe and associated with low organ toxicity and effective immunosupression for the stable engraftment in FA patients undergoing SCT with matched related donors. Fanconi anemia (FA) is a rare autosomal recessive syndrome of progressive marrow failure, constitutional physical abnormalities of skin, skeleton, kidneys, and cancer susceptibility. 1 FA cells are characterized by chromosomal instability and marked DNA hypersensitivity to crosslinking alkylating agents such as cyclophosphamide or platinum derivatives. 2,3 This hypersensitivity forms the basis for the confirmatory tests for FA with diepoxybutane (DEB), nitrogen-mustard, and others. Bone marrow failure remains the main cause of mortality among the FA patients, followed by complications of stem cell transplantation (SCT) and myeloid and nonmyeloid malignancies. Allogeneic hematopoietic SCT from unaffected sibling donors or suitably matched unrelated donors remains the only curative option for the correction of hematological abnormalities in FA patients. In the past, the use of highdose cyclophosphamide and ionizing radiation in preparative regimens often resulted in excessive organ toxicity and death in the early post transplant period. Low-doses of cyclophosphamide (20-40 mg/kg) combined with a 4-6 Gy of thoraco-abdominal or total body irradiation results in reduced toxicity and substantially improves the outcome for the FA patients transplanted from HLA-matched donors. 4 However, reduced doses of cyclophosphamide may not kill DEB-resistant naturally reverted T-lymphocytes, thereby increasing the risk of rejection in mosaic recipients of unrelated grafts (J Wagner, unpublished). Moreover, longterm follow-up clearly shows that these protocols are still associated with significant regimen-related toxicity and high risk of secondary malignant transformations. 5 More specifically, high incidence and severity of acute and chronic graft-versus-host disease (GVHD) in FA patients, who received cyclosporine alone as the GVHD prophylaxis, significantly contributed to transplantation-related morbidity and mortality, in addition favoring development of late epithelial cancers in irradiated patients. These obs...

Summary. A retrospective analysis of cyclosporine A (CsA) monotherapy administered to 66 children with aplastic anaemia (AA) at a single centre was carried out. The study was conducted on 66 children (F34/M32) with a median age of 10´5 years. 30 children (45%) achieved complete or partial remission within a median of 8 weeks. The response rate was 3/19 (16%) in cases of very severe aplastic anaemia (vSAA), 16/34 (47%) in severe aplastic anaemia (SAA) and 11/13 (85%) in nonSAA. 10 remitters (33%) relapsed after CsA cessation or dose tapering and six of those responded again on CsA alone. The only variable predictive for response was granulocyte count before treatment. The actuarial probability of survival was 51% at 4 years (85% in moderate AA, 50% in SAA and 32% in vSAA). The optimal dosage of CsA has yet to be established.

In spite of lower numerical increase in PLT count, the hemostatic efficacy and safety of PR PLT transfusions was comparable with the control group. Adverse event rates did not differ between groups, but the sample size was relatively small.

The purpose of the study was to create a certification of the service of pediatric hematology-oncology of the constituent entities of the Russian Federation (RF) using infographics and digitalization. Materials and methods. For the first time, infographic maps were used as a tool for certification, as a way of visualizing a large amount of data on the organization of pediatric hematology-oncology service in each subject of the RF. They allowed to show the specifics of the medical and organizational structure of the profile service in the subjects of the RF, their interaction with other subjects and federal centers. Processing of the cartographic image of the subject of the RF was carried out in the Adobe Illustrator graphic editor. A modifiable tool for creating infographic maps created in the MS Power Point program, with saving the results in PDF and PNG formats. Results. In the course of the study, a “Passport of Service” was prepared, containing infographic maps of 77 constituent entities of the RF. The maps in a graphic form provide information on resources available in the subject of the RF: a medical organization of the 3rd level with bed structure and the possibility of visualization, its personnel; the number of multicenter protocols and clinical guidelines used in the subject of the RF; logistics of patients, taking into account the territorial distance from the specialized beds, the presence in the region of the station/department of blood transfusion; the possibility of radiotherapy in the region; the presence of institutions where they provide palliative and rehabilitation assistance; the name of the federal and regional specialized centers, with whom the interaction is carried out, the well-established telemedicine consultation system with the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. The database of contacts of the main freelance children’s specialists of hematologists and oncologists of the RF was also updated. Conclusion.Thus, data were collected for visual display and to create specific conditions for developing optimal patient routing schemes and forming proposals for optimizing the service. The obtained “Passport of the Children’s Hematology-Oncology Service of the Russian Federation” can be used in the work of hospitals, scientific centers and professional communities.