On August 22–23, 2025, the 13th Lu Daopei Hematology Conference was held in Beijing, jointly organized by the Beijing Health Promotion Association and the Guangzhou Kapok Oncology and Rare Disease Foundation, and hosted by the Beijing Lu Daopei Hematology Institute. The conference brought together global leaders in hematology and focused on hematopoietic stem cell transplantation, cellular therapy, and precision treatment of hematologic malignancies. With more than a thousand participants, the event provided a high-level and in-depth academic platform. During the meeting, Professor Jianping Zhang presented on “Transplantation Experience in Pediatric Severe Aplastic Anemia (SAA) at Lu Daopei.” Oncology Frontier – Hematology frontier invited Professor Zhang to elaborate on the core clinical issues, offering practical guidance and theoretical reference for optimizing transplant management strategies.PART 1
Your center has accumulated extensive experience with pediatric severe aplastic anemia. Compared with traditional immunosuppressive therapy, what key standards guide your team’s selection of transplant indications? Could you also comment on timing and how it affects long-term survival?
Professor Jianping Zhang: Current domestic and international guidelines consistently recommend allogeneic hematopoietic stem cell transplantation (allo-HSCT) as one of the first-line options for pediatric SAA, with immunosuppressive therapy (IST) also being a first-line approach. Indication selection must consider whether the patient harbors gene mutations associated with inherited bone marrow failure syndromes. Patients with such mutations typically respond poorly to IST and rarely achieve durable hematologic remission; for these cases, allo-HSCT is generally preferred. For other patients in good general condition, the choice between transplantation and IST should be made jointly with the family through shared decision-making.
Donor selection and timing are critical. Ideally, transplantation should be performed as early as possible after diagnosis, particularly avoiding periods of uncontrolled active infection. If a patient fails to achieve a satisfactory response after three months of IST, a switch to allo-HSCT should be made promptly. Data clearly show that delaying transplantation reduces success rates.
Some patients present with severe infections at diagnosis. If infections cannot be controlled despite 2–3 weeks of intensive antimicrobial therapy, allo-HSCT should also be considered without delay. Traditionally, active infection was viewed as a contraindication to transplantation because of increased risks. However, in SAA, failure of hematopoietic recovery makes infection control nearly impossible. Restoring hematopoiesis and immune function through transplantation can actually provide the foundation for infection resolution and ultimately improve survival outcomes.
PART 2
How does your center balance efficacy and reduced toxicity when designing conditioning regimens for children with SAA? Are there any specific strategies tailored to pediatric patients?
Professor Jianping Zhang: Since SAA is a benign hematologic disorder, therapeutic goals extend beyond long-term survival to encompass quality of life, including control of graft-versus-host disease (GVHD), prevention of organ damage, and protection of future fertility. Conditioning regimens must therefore strike a careful balance between efficacy and safety.
For patients in acute stages with profound marrow failure, we aim to minimize use of agents with significant marrow or gonadal toxicity. In patients with residual hematopoietic function, myeloablative conditioning is still necessary to ensure engraftment, often with regimens such as busulfan-based protocols. In addition, substituting total marrow irradiation (TMI) for total body irradiation (TBI) may reduce adverse effects on growth, development, and fertility in children.
Donor choice also requires careful consideration. Patients with germline mutations in marrow failure–related genes face higher risks of relapse if transplanted from haploidentical family donors sharing the same background. In such cases, if no matched sibling or unrelated donor is available, unrelated cord blood transplantation becomes an important option. Current evidence shows that cord blood transplantation achieves survival outcomes comparable to haploidentical transplantation and may offer advantages in GVHD-free survival.
PART 3
With the continuous evolution of transplantation, has your center introduced any new techniques or concepts for pediatric patients? How have these innovations changed outcomes?
Professor Jianping Zhang: Advances in transplantation have steadily refined treatment strategies for pediatric SAA. Modern genetic testing has improved donor matching accuracy. When no germline marrow failure mutation is present, both matched sibling and haploidentical donors may be considered.
If such mutations are detected, however, related donors with shared backgrounds should be avoided, making unrelated cord blood transplantation a valuable alternative. Historically, cord blood transplantation in SAA was approached cautiously due to concerns over engraftment failure. Yet current data suggest that cord blood transplantation can outperform haploidentical transplantation in some respects, including lower rates of acute and chronic GVHD and better long-term quality of life.
Nevertheless, limitations remain. Neutrophil engraftment is typically delayed by 4–5 days and platelet recovery by 1–2 weeks compared with haploidentical transplantation. Viral infections, particularly Epstein–Barr virus (EBV), also occur more frequently after cord blood transplantation. Fortunately, advances in antiviral therapies have mitigated some of these risks.
Looking ahead, preventing EBV-related complications such as post-transplant lymphoproliferative disorder (PTLD) will be a crucial area of progress. Optimizing EBV control strategies could further expand the role of cord blood transplantation in treating SAA.
