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Isolation and Transplantation of Hematopoietic Stem Cells HSCs
Published on: February 25, 2007
Haematopoietic Stem Cell Transplantation (HSCT) for Primary Immune System Disorders in Children: A Single Centre
Asghar Ali Kerio1, Tariq Azam Khattak1, Tariq Ghafoor1
1Department of Clinical Hematology, Armed force Bone Marrow Transplant Centre / National Institute of Bone Marrow Transplant, Combined Military Hospital, Rawalpindi, Pakistan.
Insights
Hematopoietic stem cell transplantation (HSCT) outcomes in children with primary immune system disorders (PID) show a 71.4% overall survival. Fully matched HSCT improved survival compared to haplo-identical HSCT, with graft failure being the main cause of mortality.
Area of Science:
- Pediatric Hematology and Immunology
- Transplant Medicine
Background:
- Primary immune system disorders (PID) encompass a range of conditions affecting immune function.
- Hematopoietic stem cell transplantation (HSCT) is a critical treatment for severe PIDs.
- Understanding HSCT outcomes in pediatric PID is vital for improving patient management.
Purpose of the Study:
- To evaluate the outcomes of allogeneic HSCT in children diagnosed with primary immune system disorders (PID).
- To identify factors influencing survival and complications post-HSCT in this pediatric population.
Main Methods:
- A descriptive cross-sectional study was conducted at the Armed Forces Bone Marrow Transplant Centre (AFBMTC) / National Institute of Bone Marrow Transplant (NIBMT) in Pakistan.
- Data from 42 children (<12 years) with PID undergoing allogeneic HSCT between October 2012 and December 2021 were analyzed.
- Outcomes, including engraftment, complications (febrile neutropenia, mucositis, SOS/VOD, GVHD, CMV reactivation), mortality, and overall survival (OS) at 180 days, were assessed.
Main Results:
- Haemophagocytic lymphohistiocytosis (HLH) and severe combined immunodeficiency (SCID) were the most frequent diagnoses.
- Overall survival (OS) was 71.4%, with higher survival rates (80.6%) observed in patients receiving HSCT from fully HLA-matched donors compared to haplo-matched donors.
- Common complications included febrile neutropenia (73.8%), acute GVHD (40.4%), and graft failure (11.9% of total patients, leading cause of death).
Conclusions:
- Allogeneic HSCT is a viable treatment for pediatric PIDs, with HLH and SCID being the most common indications.
- Graft failure resulting in neutropenic sepsis was the primary cause of mortality.
- Outcomes favor HSCT from fully HLA-matched donors over haplo-identical donors, highlighting the importance of donor selection.
Objective:
To determine the outcomes of allogeneic HSCT in children with primary immune system disorders (PID).
Study Design:
Descriptive Cross-sectional study. Place and Duration of the Study: Armed Forces bone marrow transplant centre / National Institute of Bone Marrow Transplant (AFBMTC / NIBMT), Rawalpindi, Pakistan, from October 2012 to December 2021.
Methodology:
Data of all cases undergoing HSCT for immune system disorders were analysed for variables affecting outcome and overall survival in the first 180 days after allogeneic HSCT. All patients presenting to AFBMTC / NIBMT with PID, age <12 years. Patients with organ dysfunction secondary to repeated infections were excluded from the study. Data of all patients and their donors undergoing HSCT for immune system disorders were analysed for variables affecting outcome and overall survival in the first 180 days after allogeneic bone marrow transplant. Neutrophil engraftment was defined as absolute neutrophil count ≥0.5 × 109/L for 3 consecutive days, while platelet engraftment as platelet count ≥20 × 109/L without platelet transfusion for one week. Overall survival (OS) was taken as time from the date of HSCT till day + 180 post-transplant.
Results:
A total of 42children including 29 boys and 13 girls underwent HSCT for PID. The mean age was 2.1±2.8 years. Underlying diagnosis was haemophagocytic lymphohistiocytosis (HLH), severe immune deficiency (SCID), leukocyte adhesion defect (LAD), X-linked agammaglobulinemia, chronic granulomatous disease (CGD) and Job's syndrome in 18 (42.9%), 16 (38.1%), 3(7.1%), 2 (4.8%), 2 (4.8%) and 1 (2.4%) patients respectively. Thirty-one (73.8%) children had fully HLA-matched donors while 11 (26.2%) had haplo-matched donors. Major immediate post-transplant complications were febrile neutropenia, mucositis and SOS/VOD in 31 (73.8%), 9 (21.4%) and 4 (10.0%) cases, respectively. Eight (19.0%) had CMV reactivation, acute GVHD was seen in 17 (40.4%) cases, while 1 (2.3%) case had chronic GVHD. Twelve (28.6%) patients died, out of which 5 had graft failure, 3 had VOD, 2 had pneumonia, 1 had severe GVHD, and 1 died due to seizures. Overall survival (OS) in this study was 71.4% with survival reaching up to 80.6% in fully matched HSCT.
Conclusion:
HLH and SCID were the commonest immune disorders requiring HSCT. Graft failure leading to neutropenic sepsis was the commonest cause of mortality. OS was better in fully matched HSCT as compared to haplo-identical HSCT.
Key Words:
Immune deficiency, Severe combined immunodeficiency, Haematopoietic stem cell transplantation.
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