Initiation of Haploidentical Stem Cell Transplantation With Post-Transplant Cyclophosphamide in Children: A
Syed Ibrahim Bukhari1, Javeria Saeed2, Zehra Fadoo1
1Department of Oncology, Aga Khan University, Karachi, Pakistan.
Insights
Haploidentical hematopoietic stem cell transplant (HSCT) offers a curative option for pediatric patients lacking bone marrow registries. This study highlights challenges like graft failure and infections, impacting overall survival in this cohort.
Area of Science:
- Pediatric Hematology
- Immunology
- Transplantation Medicine
Background:
- Haploidentical hematopoietic stem cell transplant (HSCT) is a vital curative treatment, particularly in regions lacking established bone marrow registries.
- This study focuses on the application and outcomes of haploidentical HSCT in pediatric patients.
Purpose of the Study:
- To present the clinical experience and outcomes of haploidentical HSCT in pediatric patients.
- To identify challenges and factors influencing survival in this specific patient population.
Main Methods:
- A retrospective analysis of pediatric patients (≤18 years) who underwent haploidentical HSCT between January 2017 and December 2022.
- Data collected included patient demographics, disease types, donor information, stem cell source, engraftment times, complications, and survival outcomes.
Main Results:
- The cohort comprised 20 pediatric patients, with 70% having malignant diseases.
- Graft-versus-host disease (GVHD) occurred in 60% (acute) and 25% (chronic). Common complications included infection/sepsis (70%) and cytomegalovirus reactivation (70%).
- Overall survival was 40%, relapse-free survival was 50%, and treatment-related mortality was 35%. Primary graft failure was significantly higher in benign conditions (p=0.003).
Conclusions:
- Haploidentical HSCT outcomes in this pediatric cohort are comparable to those in similar low-middle-income countries.
- Graft-versus-host disease, sepsis, and cytomegalovirus infection pose significant challenges.
- Graft failure is a critical concern in nonmalignant diseases, and relapse remains high for leukemias.
Background:
Haploidentical hematopoietic stem cell transplant (HSCT) is a curative treatment especially for countries where bone marrow registries are nonexistent. We present our experience with haploidentical HSCT in pediatric patients.
Methods:
Retrospective data collected and analyzed for patients ≤18 years, from January 2017 to December 2022.
Results:
The cohort consisted of 20 patients with median age at transplant of 61.5 (IQR: 124) months. Fourteen (70%) were malignant and 6 (30%) were benign diseases. Donors were father in majority (9/20; 45%). Stem cell source was peripheral blood 8, marrow 8, and combined 4. c-specific antibodies were positive in 6 (30%). Median CD34 cell dose infused: 9.35 × 106/kg. Median engraftment time: 15 (IQR: 17) days. Acute and chronic graft-versus-host disease (GVHD) occurred in 12/20 (60%) and 5/20 (25%), respectively. Complications included infection/sepsis (14/20; 70%), cytomegalovirus reactivation (14/20; 70%), sinusoidal obstruction syndrome (1/20; 5%), primary graft failure (PGF) (6/20; 30%), and secondary graft failure (4/20; 20%). PGF was more common in benign conditions (p = 0.003) and less prevalent in cases with aGVHD (p = 0.007). aGVHD was more common in malignant conditions (p = 0.007). Overall survival (OS), relapse-free survival (RFS), and treatment-related mortality (TRM) were 40%, 50%, and 35%, respectively. Median time of survival and relapse were 8 (IQR: 15) and 9 (IQR: 13) months, respectively.
Conclusion:
OS was comparable to that of other low-middle-income countries. GVHD was a major challenge, along with sepsis and CMV infection. Half of the leukemias relapsed. Graft failure was a major concern in nonmalignant diseases.
Related Concept Videos
01:22Bone Marrow Sampling and Transplants
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
01:21Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
01:24Tissue Transplantation
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...