Long-Term Outcome Prediction After Immunosuppressive Therapy for Severe Aplastic Anemia in Childhood by Machine Learning Methods

2020 ◽  
Author(s):  
Lixian Chang ◽  
Mingchen Yan ◽  
Jingliao Zhang ◽  
Binghang Liu ◽  
Ye Guo ◽  
...  
Keyword(s):  
Machine Learning ◽  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Outcome Prediction ◽  
Severe Aplastic Anemia ◽  
Learning Methods ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Machine Learning Methods

scholarly journals Very long-term follow-up of aplastic anemia treated with immunosuppressive therapy or allogeneic hematopoietic cell transplantation

2020 ◽  
Vol 99 (11) ◽  
pp. 2529-2538
Author(s):  
Beatrice Drexler ◽  
Felicitas Zurbriggen ◽  
Tamara Diesch ◽  
Romaine Viollier ◽  
Joerg P. Halter ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Immunosuppressive Therapy ◽  
Hematopoietic Cell Transplantation ◽  
Clonal Evolution ◽  
University Hospital ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Long Term Survivors

Abstract Introduction Since the 1970s outcome of aplastic anemia (AA) patients has improved significantly due to the introduction of immunosuppressive therapy (IST) and allogeneic hematopoietic transplantation (HCT). However, patients may suffer from persistent disease, relapse, clonal evolution, graft-versus-host disease and other late effects. Here, we analyse very long-term outcome of all AA patients at our institution comparing not only survival, but also response status and complications. Methods Patient charts of all 302 AA patients treated between 1973 and 2017 at the University Hospital Basel, Switzerland, were retrospectively analysed. Results First line treatment was IST in 226 (75%) and HCT in 76 (25%) patients. Overall survival at 30 years was similar in patients treated initially by HCT and IST (44% (±14%), and 40% (± 9%) respectively, with better results in more recent years. Partial and no response occurred more frequently after IST, relapse incidence after IST was 24 %, whereas non-engraftment and graft failure was documented in 15 patients (19 %) after HCT. Clonal evolution to myelodysplastic syndrome / acute myeloid leukemia was 16 % at 25 years in IST patients, 1.3 % in HCT patients, iron overload (18 versus 4 %, p = 0.002) and cardiovascular events (11 versus 1 %, p=0.011) occured significantly more often in IST than HCT treated patients. The majority of long-term survivors, 96% of those alive at 25 years, were in complete remission at last follow up, irrespective of the initial treatment modality. Conclusion Very long term survivors after AA are those with stable hematopoietic recovery.

scholarly journals Long-term outcome of pregnancy complicating with severe aplastic anemia under supportive care

2017 ◽  
Vol 56 (5) ◽  
pp. 632-635 ◽  
Author(s):  
Kuan-Ju Chen ◽  
Yao-Lung Chang ◽  
Horng Chang ◽  
Shen-Yuan Su ◽  
Hsiu-Huei Peng ◽  
...  
Keyword(s):  
Supportive Care ◽  
Aplastic Anemia ◽  
Severe Aplastic Anemia ◽  
Term Outcome ◽  
Long Term Outcome

scholarly journals O9.7. INDIVIDUALIZED LONG-TERM OUTCOME PREDICTION OF PSYCHOSIS IN AN OBSERVATIONAL STUDY: A MACHINE LEARNING APPROACH

2018 ◽  
Vol 44 (suppl_1) ◽  
pp. S101-S102 ◽  
Author(s):  
Jessica De Nijs ◽  
Daniel P J van Opstal ◽  
Ronald J Janssen ◽  
Wiepke Cahn ◽  
Hugo Schnack ◽  
...  
Keyword(s):  
Machine Learning ◽  
Observational Study ◽  
Outcome Prediction ◽  
Learning Approach ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Machine Learning Approach

Use of G-CSF in Patients with Severe Aplastic Anemia Treated with ATG and Cyclosporine Increases Neutrophils and Decreases Infection Rates and Hospitalization Days but Does Not Improve Long-Term Outcome: Results of a Prospective, Randomized Clinical Trial of the EBMT.

Blood ◽  
2009 ◽  
Vol 114 (22) ◽  
pp. 496-496 ◽  
Author(s):  
AndrÉ Tichelli ◽  
Hubert Schrezenmeier ◽  
Gerard Socié ◽  
Judith Marsh ◽  
Andrea Bacigalupo ◽  
...  
Keyword(s):  
Aplastic Anemia ◽  
Working Party ◽  
Response Rates ◽  
Severe Aplastic Anemia ◽  
Controlled Study ◽  
Term Outcome ◽  
Hematopoietic Stem ◽  
Long Term Outcome ◽  
Treatment Groups

Abstract Abstract 496 Immunosuppression (IS) with ATG and Cyclosporine (CSA) is the treatment of choice for patients with severe aplastic anemia (AA) not eligible for hematopoietic stem cell transplantation. The role of additional growth factors such as G-CSF on long-term outcome is still a matter of debate. The Aplastic Anemia Working Party of the EBMT conducted a randomized controlled study comparing IS with ATG and CSA with or without G-CSF and with or without early retreatment at day 120 in case of nonresponse. The study was designed to identify a difference of 10% for overall survival (OS) and event free survival (EFS). Three hundred forty subjects were to be enrolled. From 2002 to 2008, 205 patients were randomized, but 13 had to be excluded (1 incorrect diagnosis; 12 no follow-up data); 192 were evaluable for analysis (95 with G-CSF; 97 without G-CSF). The median age of the patients was 46 years (2-81), 95 (49%) were males, 69 (36%) had very severe AA. There was no difference between both groups in respect of age, sex, severity of the AA, and number of transfusions before treatment. Horse ATG (Lymphoglobulin) was administered (15mg/kg/BW/d ×5 days), and CSA given orally at a dose of 5mg/kg/d. Patients randomized to receive G-CSF were given glycosylated rHuG-CSF from day 8 to 120 (150mg/m2/d, sc). OS at 6 years was 75%, it was 82% for patients with severe AA and 66% for patients with very severe AA (P=0.001). Survival decreased with increasing age from 100% (age <20years) and 92% (20-40 years) to 71% (40-60 years) and 56% (>60 years) (P<0.001). There was no difference in OS (P=0.64) and EFS, defined by death, need for transplantation, relapse and non response as events (P=0.358) between the study arms at 6 years. This was the case for the entire cohort as well as when stratified according to age and AA severity (Table). The median number of neutrophils was significantly higher between day 30 and 240, in the treatment group but this difference did not persist to day 360, when G-CSF was stopped. There were fewer infections (36% no G-CSF; 24% with G-CSF; P=0.006), and less days of hospitalization during the first 90 days (P=0.03) in the group of patients with G-CSF, mainly for patients with very severe AA. During the study period 44 patients died. The most common cause of death was infection (55%).There was no difference in deaths and causes of death between both treatment groups. However, there were more late deaths (>3 years) in the G-CSF group (P=0.01). There was no difference in respect of response rates between both groups. Overall 73% of patients with and 62% without G-CSF did respond to IS (P=0.488). Response rates at days 30, 60, 90 120, 150, 180, 240 and 360 were similar between the treatment groups. Fifty seven patients did not respond to first-line therapy, and 31 patients relapsed during the first year of treatment, without any difference between both groups. In conclusion, G-CSF given together with standard IS increases neutrophil counts, and decreases rate of infections and days of hospitalization, mainly in patients with very severe AA. G-CSF does not improve long-term outcome and has no impact on OS, EFS, remission, death and relapse rates. G-CSF may decrease infection risks when used as an adjunct to IS therapy. This has to be weighed against possibly higher risks of MDS/AML, as suggested by previous studies. Disclosures: Schrezenmeier: Alexion: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding.

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