Current incidence, severity, and management of veno-occlusive disease/sinusoidal obstruction syndrome in adult allogeneic HSCT recipients: an EBMT Transplant Complications Working Party study.

The current incidence, diagnostic policy, management, and outcome of VOD/SOS at EBMT centers were studied. All centers that had performed allogeneic HSCTs in adult patients within one defined year were invited to the study. Seventy-one centers participated with a total of 2886 allogeneic transplantations and 93 cases of VOD/SOS in 2018. The cumulative incidence of VOD/SOS at day 21 was 1.8% and at day 100 2.4%. Of 67 cases with detailed data, 52 were classical and 15 (22%) late onset (>day 21). According to the EBMT criteria, 65/67 patients had at least two VOD/SOS risk factors. The severity grades were: mild 0, moderate 3, severe 29, very severe 35. Fifty-four patients were treated with defibrotide. VOD/SOS resolved in 58% of the patients, 3/3 with moderate, 22/28 with severe, and 12/33 with very severe grade (p < 0.001). By day 100, 57% of the patients were alive; 3/3 with moderate, 22/29 with severe, and 13/35 with very severe VOD/SOS (p = 0.002). In conclusion, the incidence of VOD/SOS was low. Severe and very severe grades dominated. Very severe grade predicted poor outcome compared to severe grade further supporting the concept of early diagnosis and treatment to avoid a dismal outcome.

Association of Country-Specific Socioeconomic Factors With Survival of Patients Who Experience Severe Classic Acute Graft-vs.-Host Disease After Allogeneic Hematopoietic Cell Transplantation. An Analysis From the Transplant Complications Working Party of the EBMT.

Acute graft-vs.-host disease (aGvHD) is one of the most frequent causes of transplant-related mortality (TRM) after allogeneic hematopoietic cell transplantation (alloHCT). Its treatment is complex and costly. The aim of this study was to retrospectively analyze the impact of country-specific socioeconomic factors on outcome of patients who experience severe aGvHD. Adults with hematological malignancies receiving alloHCT from either HLA-matched siblings (n = 1,328) or unrelated donors (n = 2,824) developing grade 3 or 4 aGvHD were included. In univariate analysis, the probability of TRM at 2 years was increased for countries with lower current Health Care Expenditure (HCE, p = 0.04), lower HCE as % of Gross Domestic Product per capita (p = 0.003) and lower values of the Human Development Index (p = 0.02). In a multivariate model, the risk of TRM was most strongly predicted by current HCE (HR = 0.76, p = 0.006). HCE >median was also associated with reduced risk of the overall mortality (HR 0.73, p = 0.0006) and reduced risk of treatment failure (either relapse or TRM; HR 0.77, p = 0.004). We conclude that country-specific socioeconomic factors, in particular current HCE, are strongly associated with survival of patients who experience severe aGvHD.

Cost-benefit assessment of antifungal prophylaxis with liposomal amphotericin B in neutropenic patients.

BACKGROUND: In a recently reported randomized trial, low-dose intravenous liposomal amphotericin B (L-AmB) reduced the incidence of invasive fungal infections (20.2 vs. 4.6%, p < 0.001) in high-risk patients with hematological malignancies and prolonged neutropenia. PATIENTS AND METHODS: In the present study, we performed a retrospective cost-benefit analysis of L-AmB prophylaxis from the hospital perspective. RESULTS: Ninety-nine patients were eligible; baseline characteristics were balanced for age, sex, underlying disease, and duration of neutropenia. The mean duration of hospitalization was 42.9 days and 52.3 days in the prophylaxis arm and in patients without antifungal prophylaxis, respectively (p = 0.096). The L-AmB prophylaxis was associated with additional costs of approximately EUR 630 per patient. However, total medication costs (including L-AmB prophylaxis) were EUR 1,219 and EUR 2,815 in patients with L-AmB prophylaxis and in patients in the control arm, respectively (p < 0.001). When involving also costs for medical procedures, the L-AmB prophylaxis reaches a positive net benefit of EUR 1,094 per patient. CONCLUSIONS: Our data shows that antifungal prophylaxis, e.g. with L-AmB, can be a safe and effective strategy to reduce the frequency of invasive fungal infections in selected high-risk patients and to obtain significant cost savings for the hospital.