1.
Reduced risk of sinusoidal obstruction syndrome of the liver after Busulfan-Cyclophosphamide conditioning prior to allogeneic hematopoietic stem cell transplantation
El-Serafi, I., Remberger, M., Ringden, O., Torlen, J., Sundin, M., Bjorklund, A., Winiarski, J., Mattsson, J.
Clinical and translational science. 2019
Abstract
The aim of this study is to evaluate the incidence of sinusoidal obstruction syndrome (SOS) of the liver and the clinical outcome after hematopoietic stem cell transplantation (HSCT) based on several modifications in our protocols. We retrospectively investigated 372 patients undergoing myeloablative conditioning with oral busulfan and cyclophosphamide before allogeneic HSCT during 1990-2015. Patients' supportive care was changed in order to reduce the regimen-related toxicities. Norethisterone use was terminated in 1998, therapeutic drug monitoring of busulfan was initiated in 2000 and the use of liver supportive drugs, such as ursodeoxycholic acid (UDCA) and N-acetyl-L-cysteine (NAC), were started in 2002 and 2009, respectively. In total, 26 patients (7.0%) developed SOS at a median of 19 days after transplantation. Of these 26 patients, 20 died at a median of 119 days after HSCT and 102 days after the diagnosis of SOS. The incidence of SOS decreased over time in accordance with the improvements in supportive care. The highest incidence of SOS was during 1995-99 (16.2%) compared to 2.3% during 2010-2015. Overall survival for patients with SOS was 62%, 46% and 27% at 100 days, 1 year and 5 years after HSCT respectively compared to 92%, 77% and 66% for those who did not develop SOS (P<0.001). In conclusion; the incidence of SOS and related deaths were significantly decreased over the last years. Our institution pursues massive preventative and personalized measures for SOS. This strategy may also be applicable in other conditioning protocols in order to reduce the incidence of SOS and hence, improve the clinical outcome.
2.
Granulocyte transfusions could benefit patients with severe oral mucositis after allogeneic hematopoietic stem cell transplantation
Berglund, S., Watz, E., Remberger, M., Garming Legert, K., Axdorph-Nygell, U., Sundin, M., Uhlin, M., Mattsson, J.
Vox sanguinis. 2019
Abstract
BACKGROUND AND OBJECTIVES Mucositis is a common complication after allogeneic hematopoietic stem cell transplantation (HSCT), and is caused by a combination of conditioning-induced mucosal damage and severe neutropenia. The symptoms include oral and abdominal pain, inability to swallow food and fluids, and severe diarrhoea. Severe mucositis is associated with increased risk of Graft-versus-Host disease and infection. Granulocyte transfusions (GCX) could be a treatment option, and our objective was to study its feasibility and potential benefits. MATERIAL AND METHODS This retrospective, single-centre study included 30 patients receiving GCX because of severe oral mucositis after HSCT during 2005-2017. Clinical outcome, response to GCX, change in opiate administration and adverse events were studied. RESULTS Twenty-seven patients received GCX from donors pre-treated with steroids and G-CSF, and three from donors pre-treated with steroids only. Overall response was 83% (24/29 evaluable patients). Fifteen patients reached a complete response. In 14 of 24 responders, a reduction of the administration of opiate pain relief was seen. In eight patients this reduction was ≥50% of the dose. Adverse events (AEs) were reported in 14 cases, and were mild to moderate, and well manageable with symptomatic treatment. No life-threatening or fatal AEs were recorded. CONCLUSIONS These results indicate that GCX could be a safe and effective treatment for oral mucositis after HSCT with the potential to reduce the necessity of opiate analgesic treatment in this disorder. No severe AEs were seen in this study, but the risk for severe pulmonary AEs after GCX needs to be considered.