scholarly journals Evolution of interstitial lung disease one year after hematopoietic stem cell transplantation or cyclophosphamide for systemic sclerosis

2020 ◽  
Author(s):  
Jacopo Ciaffi ◽  
Nina M. van Leeuwen ◽  
Maaike Boonstra ◽  
Lucia J.M. Kroft ◽  
Anne A. Schouffoer ◽  
...  
2014 ◽  
Vol 108 (10) ◽  
pp. 1525-1533 ◽  
Author(s):  
Frédéric Schlemmer ◽  
Sylvie Chevret ◽  
Gwenaël Lorillon ◽  
Cédric De Bazelaire ◽  
Régis Peffault de Latour ◽  
...  

2021 ◽  
Vol 11 (7) ◽  
pp. 600
Author(s):  
Paul Gressenberger ◽  
Philipp Jud ◽  
Gabor Kovacs ◽  
Sonja Kreuzer ◽  
Hans-Peter Brezinsek ◽  
...  

Systemic sclerosis (SSc) is an intractable autoimmune disease characterized by vasculopathy and organ fibrosis. Autologous hematopoietic stem cell transplantation (AHSCT) should be considered for the treatment of selected patients with rapid progressive SSc at high risk of organ failure. It, however, remains elusive whether immunosuppressive therapies such as rituximab (RTX) are still necessary for such patients after AHSCT, especially in those with bad outcomes. In the present report, a 43-year-old man with diffuse cutaneous SSc received AHSCT. Despite AHSCT, SSc further progressed with progressive symptomatic heart failure with newly developed concomitant mitral and tricuspid valve insufficiency, thus the patient started on RTX 8 months after AHSCT. Shortly after initiation of RTX, clinical symptoms and organ functions ameliorated subsequently. Heart valve regurgitations were reversible after initiation of RTX treatment. Currently, the patient remains in a stable condition with significant improvement of clinical symptoms and organ functions. Reporting about therapies after AHSCT in SSc is a very important issue, as randomized controlled trials are lacking, and therefore this report adds to evidence that RTX can be considered as a treatment option in patients with SSc that do not respond to AHSCT.


Blood ◽  
2007 ◽  
Vol 110 (11) ◽  
pp. 1996-1996
Author(s):  
Julia A. LeMaistre ◽  
Carlos Bachier ◽  
Brad Smith ◽  
C.F. LeMaistre ◽  
Paul J. Shaughnessy

Abstract The development of intravenous busulfan (Bu) allowed for the delivery of a standard daily dose of Bu to be given in one dose instead of dividing 4 times per day. Pharmacokinetic (PK) studies of Bu support the feasibility of once daily dosing and prior clinical studies found no unexpected toxicity of once daily Bu when combined with fludarabine. Based on this data we began an institutional protocol of delivering Bu once daily follwed by standard cyclophosphamide (Cy) and allogeneic hematopoietic stem cell transplantation (HCT). Only limited data in the literature describes toxicity and clinical outcomes of once daily BuCy. We report a retrospective review of our institutional data using once daily BuCy vs 4x daily BuCy and total body irradiation (TBI)/Cy in pts who received allogeneic HCT from January 2000 to December 2006. Bu 3.2mg/kg daily × 4 days followed by Cy 60mg/kg daily × 2 days was given to 42 patients (pts). Bu 0.8mg/kg/dose given 4x daily for 4 days followed by Cy 60mg/kg was given to 15 pts. Cy 60mg/kg daily for 2 days and fractionated TBI 1200 cGy delivered over 3 days was given to 60 pts. All donors were HLA matched at A, B, C, DR, and DQ and were related/unrelated in 23/19, 11/4, and 21/39 in the once daily BuCy, 4x daily BuCy, and TBI/Cy, respectively. Significantly more pts with myeloid leukemias received a BuCy regimen and significantly more pts with lymphoid malignancies received TBI/Cy. Median follow up for all pts was 370 days. VOD developed in 2 pts in the once daily BuCy group, 1 pt in the 4x daily BuCy group, and in no pts in the TBI/Cy group. Acute GVHD grade II-IV occured in 33% of the once daily BuCy pts, 53% of the 4x daily BuCy pts, and 32% of the TBI/Cy pts. Estimated actuarial transplant related mortality (TRM) and survival are described in the table below. There was no statistical difference in TRM or survival between the once daily and 4x daily BuCy groups or the total BuCy group and TBI/Cy group. The once daily BuCy group had significantly less TRM than the TBI/Cy group at 100 days (p=0.04) and at 1 year (p=0.01). The once daily BuCy group also had significantly better survival at one year compared to the TBI/Cy group (p=0.01), but this became non-significant at 3 years. The significant differences in the pt populations treated in the BuCy and TBI/Cy groups, as well as the retrospective nature of this study, limit the ability to draw conclusions comparing the groups. However, this review does demonstrate once daily BuCy and allogeneic HCT is well tolerated with no unexpected TRM, and provides good long term survival in pts with myeloid malignancies. Survival and TRM 1xBuCy (95%CI) 4xBuCy (95%CI) Cy/TBI (95%CI) 100 day TRM 7% (1–16%) 21% (7–51) 22% (15–35) 1 year TRM 21% (11–38) 37% (17–69) 45% (33–59) 1 year Survival 70% (53–82) 47% (21–69) 49% (35–61) 3 year Survival 43% (24–59) 27% (8–50) 37% (24–49)


Blood ◽  
2011 ◽  
Vol 118 (21) ◽  
pp. 3087-3087
Author(s):  
Mauricette Michallet ◽  
Mohamad Sobh ◽  
Stephane Morisset ◽  
Marie Y. Detrait ◽  
Helene Labussiere ◽  
...  

Abstract Abstract 3087 Introduction Allogeneic hematopoietic stem cell transplantation (allo-HSCT) represents the only potential to cure wide types of hematological diseases. A patient has 30% of chance to find a HLA-identical sibling donor while the rest of patients should find an alternative unrelated donor. The use of 10/10 HLA matched unrelated transplants has been used as a main alternative and with its unavailability, when available, a 9/10 HLA mismatched unrelated transplant has been used. The outcome of this last mismatched transplant is not very clear and its use according to patient and disease conditions has not been well defined yet. Aims To evaluate the outcome of allo-HSCT from 9/10 HLA mismatched unrelated donors compared to those from 10/10 HLA identical unrelated donors and siblings; and to define which category of patients can benefit the more in each alternative. Material and methods We have retrospectively studied the outcome of 213 patients who received allo-HSCT for different hematological malignancies, 121 (57%) from HLA identical siblings, 63 (29%) from 10/10 HLA identical unrelated donors and 29 (14%) from 9/10 HLA mismatched unrelated donors treated during the same period of time between 2006 and 2011 at our institution. In the mismatched group, 12 patients had the mismatch at HLA-A locus, 7 at the HLA-B, 7 at the HLA-C and 3 at the HLA-DQ. Characteristics between the 3 groups were comparable except for: disease type between the 2 unrelated groups, sex-matching, CMV-matching and ABO-matching. The different characteristics are detailed in Table 1. Results After HSCT, engraftment was significantly lower in the 9/10 HLA group (90%) than in the 10/10 HLA group (95%) than in the sibling group (99%), (p=0.03); the cumulative incidence of acute GVHD ≥2 at 3 months was 32% (23–41), 20% (15–26) and 27% (23–32) respectively; the cumulative incidence of extensive chronic GVHD at one year was 21% (13–30), 9% (5–13) and 17% (14–21) for the 3 groups respectively. After a median follow-up of 8 months (0–54) in the 9/10 HLA group, 10 months (0–60) in the 10/10 HLA group and 18 months in the siblings group, the median overall survival (OS) was 10 months (5–21), 18 months (11-NR) and 60 months (31-NR) respectively with a 2-years probability of 19% (8–44), 43% (31–59) and 63% (54–74) respectively. There was a higher but not significant relapse incidence at one year in the 9/10 HLA group compared to other groups while the transplant related mortality was significantly higher with a cumulative incidence at 1 year of 45% (35–55), (p<0.001) (Table1-results). In multivariate analysis, OS was negatively affected by unrelated donors [9/10 HR=5 (2.7–10), p=0.0001; 10/10 HR=2 (1.2–4), p=0.01], female donors [HR=2 (1.4–4), p=0.03] and disease status < CR1 or <chronic phase (CP) 1 [HR=3 (1.4–6), p=0.003]; while the TRM was negatively affected by unrelated donors [9/10 HR=9 (4–20), p<0.001; 10/10 HR=4 (1.2–10), p=0.03], female donors [HR=3 (1.2–7); p=0.01] and ABO minor incompatibility [HR=2.5 (1.2–5), p=0.01]. The funnel plot showing the adjusted TRM according to all covariates and comparing to the global population death rate, shows that the 9/10 HLA group has the worse TRM independently of any other factor. Conclusion We showed that allo-HSCT from 9/10 HLA mismatched unrelated donors have a significantly worse OS than those from matched unrelated donors and siblings; this was mainly due to an increased TRM in this group. Patients in first CR or CP could benefit the more from matched or 9/10 unrelated allo-HSCT while the use of transplants from 9/10 HLA unrelated donors in patients not in CR1 or CP1 should be limited to clinical trials. In view of these results, we should consider and evaluate the use of cord blood as an alternative source of transplant according to patient and disease conditions. Disclosures: No relevant conflicts of interest to declare.


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