Retrospective evaluation of transplant patients followed in Çukurova University Faculty of Medicine paedatric bone marrow transplantation center

Objective: Hematopoietic Stem Cell Transplantation (HSCT) is a treatment method, remaining a dangerous procedure with many possible complications including death, has been performed for the patients with malignant and hematological diseases with increasing indications in recent years, and leading to healthy hematological and immunological restructuring. In this study, we aimed to evaluate the demographic data, donor characteristics, pre-transplant preparation regimens, duration of engraftment, post-transplant complications and treatments, and survival in hematopoietic stem cell transplant patients. Material and Method: The study included 242 patients who underwent HSCT in the Pediatric Bone Marrow Transplant Unit of Çukurova University School of Medicine (CUTF) between 27 January 1993 and 24 May 2019. Patient data were collected from patient files and computer information system retrospectively. Findings: 62.4% (151 people) of the patients are boys and 37.6% (91 people) are girls. While 81.4% of the patients were Turkish citizens, 18.6% were foreign nationals. The median age of the patients was 93 months (7 months-225 months) 7 years 7 months. Transplantation was performed for 103 (42.6%) patients with malignant disease, 117 (48.3%) with hematological disease, 19 (7.8%) with immunological disease and 3 (1.2%) with metabolic disease. 103 patients were analysed in the malignant disease group. 22 (21%) were ALL, 18 (17.4) were AML, 14 (13.5%) were HL, 10 (9.7%) were MDS, 8 (7.7%) were NHL. Solid tumors including 5 (4.8%) HLH, 2 (1.9%) KML, 1 (0.9%) JMML and 18 (17.4%) neuroblastoma, 2 (1.9%) ewing sarcoma, 2 (% 1.9) rhabdomyosarcoma, 23 patients (22.3%), including rhabdomyosarcoma and 1 (0.9%) epandymoma, were diagnosed in 23 patients. Of the 117 patients in the hematological disease group, 72 (61.5%) were thalassemia, 30 (25.6%) were FAA, 8 (6.8%) were aplastic anemia, 5 (4.2%) were SCA, 1 (0.8%) were congenital dyseritropoietic anemia and 1 (0.8%) were diagnosed as diamond-blackfan anemia. Allogenic transplantation and autologous transplantation were performed in 99 (82.2%) patients and 43 (17.8%) patients respectively. Allogenic transplants were conducted from 168 (83.7%) relatives and 31 (16.3%) non-relatives. 126 (82.9%) of the patients were 10/10, 24 (15.8%) of them were 9/10 and 2 of them were 8/10 HLA compatible transplants. The stem cell sources derived form bone marrow in 150 patients (62.2%), peripheral blood in 90 patients (37.3%), umbilical cord blood in 1 patient (0.4%); In 2 patients, umblical cord blood and bone marrow were used. In the preparation regimen, myeloablative protocol was used for 175 patients (81%) and non myeloablative protocol was used for 41 patients (18.9%). While myeloid engraftment was occured in 201 (93.1%) the patients who underwent HSCT, engraftment was not developed in 15 (6.9%) patients. The average time of development of engraftment was 13 (6-44) days. Loss of engraftment occurred in 22 patients (11.6%). Early postoperative complications were seen in 149 patients (61.6%), 2 patients (0.8%) had late, and 56 patients (23.1%) had both early and late complications. Antibiotherapy was administered to 200 (97.5%) patients with bacterial infection clinic and blood cultures of 76 (37%) detected the presence of bacteria. Viral infection was observed in 109 (53.1%) of the patients after transplantation. Fungal infections were seen in 37 patients (18%), while 2 (5.4%) blood culture reproduction was detected. 55 (26.4%) patients who had early GVHD. The mean time to determine GVHD was 54 (3-207) days. Late acute GVHD was seen in 14 patients (6.7%) and chronic GVHD was seen in 4 patients (1.9%). Skin involvement was observed in 41 (75.9%) of GVHD, intestinal involvement in 34 (62.9%), and liver involvement in 10 (18.5%). 15 (27.8%) of GVHD were grade I , 17 of them (31.5%) were classified as grade II, 17 (31.5%) were classified as grade III and 5 (9.3%) were classified as grade IV. GVHH mortality was 61.1% (n = 33) (p = 0.004). The diagnosis of hemorrhagic cystitis was diagnosed in 30 patients (14.3%) and 9 patients (4.3%) were diagnosed with sinusoidal obstruction syndrome. Late complications were observed in 58 patients (28%). Second transplantation was performed in 17 patients (7%) not provided engraftment, and third transplantation (0.4%) was perfomed in 1 patient. Secondary cancer was developed in 1.2% of patients; recurrence occurred in 30%, and 39.3% were exitus after the transplantation.After the transplantion, one-year estimated survival was 64.9%, three-year survival was 52.8% and 5-year survival was 49.7%. Conclusion: Although the rate of morbidity and mortality is high after HSCT, it is generally the last healing option in many diseases. Retrospective review of the HSCT process protocols in our transport unit; identifying prognostic markers can be useful for providing guidance for future follow-up measures and creating individual stem cell transplant strategies to improve short and long term complications. Creating a risk-tailored treatment with a special long-term follow-up program can further increase HSCT success preventing secondary morbidity to improve long-term quality of life and overall survival in HSCT patients.