National Longitudinal Cohort of Hematological Diseases (NICHE)
Conditions: Multiple Myeloma, Acute Myeloid Leukemia, Hemophilia, Hemophilia A, Hemophilia B, Myelodysplastic Syndrome, MDS, Lymphoma, Leukemia, Aplastic Anemia, Bleeding Disorder, Bone Marrow Transplantation, Blood Disease Infection, Autoimmune Hemolytic Anemia, AIHA, Essential Thrombocythemia, ET, Large Granular Lymphocyte Leukemia, LGLL, Paroxysmal Nocturnal Hemoglobinuria, PNH
Sponsor: Institute of Hematology & Blood Diseases Hospital, China

 
National Longitudinal Cohort of Hematological Diseases 
- Bone Marrow Transplantation 
(NICHE- BMT) 
 
 
 
 
 
 
Applicant: Institute of Hematology & Blood Diseases Hospital 
Version: 1.0 
NCT Number: NCT04645199 
Date: 15-Dec-2020 
 
 
 

 
National 
Longitudinal Cohort of Hematological 
Diseases- 
Bone Marrow 
Transplantation (NICHE-BMT) 
 
Background 
Hematological diseases are disorders of the blood and hematopoietic organs. The 
current hematological cohorts are mostly based on single-center or multi-center cases, 
or cohorts with limited sample size in China. There is a lack of comprehensive and 
large-scale prospective cohort studies in hematology.  
 
Objectives 
The objectives of this study are to investigate the characteristics of patients received 
bone marrow transplantations and to analyze the treatment methods, prognosis and 
medical expenses of these patients in China. 
 
Subjects 
Patients who were received bone marrow transplantations in the investigating hospitals 
from February 7, 2021. 
 
Method 
The NICHE-BMT will collect basic information, diagnostic and treatment information, 
as well as medical expense information of patients from medical records. The study 
will use questionnaire to measure the exposure of patients, and prospectively follow-up 
to collect the prognosis information. 
 
 
Statistical Analysis Plan 
Statistical analyses will be performed using SAS 9.4 (SAS Institute Inc., Cary, NC, 
USA) software. For the categorical variables, the results will be presented as counts and 
percentages. The differences between groups were compared with Chi-square test or 
Fisher's exact test. For continuous variables, mean ± SD or median (P25, P75) will be 
presented according to the distributions. The differences between groups were 
compared with ANOVA or Wilcoxon rank-sum test. For time-to-event variables, the 
incidences of events were described using the Kaplan-Meier method and compared 
between groups using the log-rank test. Cox proportional hazard regression modes were 
used for multivariate analyses. A P-value of less than 0.05 was considered to indicate 
statistical significance. 
 
 

 
National Longitudinal Cohort of Hematological Diseases 
- Pediatric hematological diseases 
(NICHE- Pediatrics) 
 
 
 
 
 
 
Applicant: Institute of Hematology & Blood Diseases Hospital 
Version: 1.0 
NCT Number: NCT04645199 
Date: 9-Dec-2020 
 
 

 
National Longitudinal Cohort of Hematological Diseases- Pediatric Hematological 
Diseases (NICHE- Pediatrics) 
 
Background 
Hematological diseases are disorders of the blood and hematopoietic organs. The 
current hematological cohorts are mostly based on single-center or multi-center cases, 
or cohorts with limited sample size in China. There is a lack of comprehensive and 
large-scale prospective cohort studies in hematology.  
 
Objectives 
The objectives of this study are to investigate the incidence and risk factors of AML, 
ALL, AA, IBMFS, MPN, et al. and to analyze the treatment methods, prognosis and 
medical expenses of these pediatric patients in China. 
 
Subjects 
Pediatric patients who were diagnosed with AML, ALL, AA, IBMFS and MPN, et al. 
in the investigating hospitals from February 3, 2021. 
 
Method 
The NICHE-Pediatrics will collect basic information, diagnostic and treatment 
information, as well as medical expense information of patients from medical records. 
The study will use questionnaire to measure the exposure of patients, and prospectively 
follow-up to collect the prognosis information. 
 
 
Statistical Analysis Plan 
Statistical analyses will be performed using SAS 9.4 (SAS Institute Inc., Cary, NC, 
USA) software. For the categorical variables, the results will be presented as counts and 
percentages. The differences between groups were compared with Chi-square test or 
Fisher's exact test. For continuous variables, mean ± SD or median (P25, P75) will be 
presented according to the distributions. The differences between groups were 
compared with ANOVA or Wilcoxon rank-sum test. For time-to-event variables, the 
incidences of events were described using the Kaplan-Meier method and compared 
between groups using the log-rank test. Cox proportional hazard regression modes were 
used for multivariate analyses. A P-value of less than 0.05 was considered to indicate 
statistical significance.