The long-term quality of life (QOL) of children after hematopoietic stem cell transplantation (HSCT) has been reviewed at the presidential symposium “Supporting children after HSCT” in the 41st Annual Meeting of the Japan Society for Hematopoietic Cell Transplantation. First, I talked about the methods to manage QOL issues in HSCT cases. Second, I summarized previously published observational studies on QOL after HSCT that included more than 40 subjects. I have divided the studies into 4 categories and summarized their results: Ⅰ. cross-sectional studies with a comparison group; Ⅱ. cross-sectional studies without a comparison group; Ⅲ. longitudinal studies that evaluated the change in QOL; and Ⅳ. studies on subjects such as parents and siblings of children with HSCT. Third, I explained the main results of the Japanese cross-sectional QOL study recently conducted among children with long-term survival after HSCT. Finally, I proposed some future studies on QOL of children after HSCT.
Background: The elucidation of clinical characteristics in deceased patients is essential to improve outcomes of hematopoietic stem cell transplantation (HSCT) for refractory/relapsed hematological malignancy. Patients and Methods: We retrospectively examined 81 refractory/relapsed hematological malignancy patients treated with allogeneic HSCT (allo-HSCT) (54) and autologous HSCT (auto-HSCT) (27) in our hospital from 2006 to 2016. Results: Consistent with previous Japan Marrow Donor Program annual reports, the overall survival (OS) rate of allo-HSCT and auto-HSCT patients were 59% and 84% at five years, respectively. Among patients receiving allo-HSCT, severe regimen-related toxicity (RRT) (grade≥3) events included cardiomyopathy due to cyclophosphamide (1), idiopathic pulmonary syndrome (1), acute graft-versus-host disease (GVHD) Ⅲ-Ⅳ (3), acute-exacerbated chronic GVHD (2), engraftment failure (2), human herpesvirus-6 encephalitis (2), and fungal infection (7). Moreover, univariate analysis identified disease risk index (DRI) and non-CR status before allo-HSCT as prognostic factors of OS. Among patients receiving auto-HSCT, the severe RRT event was thrombotic microangiopathy (1). The relapse after auto-HSCT in three patients with malignant lymphoma was a serious concern. Conclusion: Our study revealed critical issues in non-CR patients and those with high/very high DRI before allo-HSCT. Furthermore, the occurrence of severe RRT indicated the need for improvements in allo- and auto-HSCT.
Pulmonary veno-occlusive disease (PVOD), a variant of pulmonary arterial hypertension, is a rare complication that can occur after hematopoietic stem cell transplantation (HSCT) and chemotherapy. PVOD clinically presents with pulmonary edema. A 21-year-old Down Syndrome female with very early relapsed acute B-cell precursor lymphoblastic leukemia underwent cord blood transplantation (HLA 5/6 antigen-matched) after achieving her third complete remission with a reduced-intensity conditioning regimen using fludarabine, melphalan and total body irradiation (4 Gy). During the conditioning chemotherapy she suffered from dyspnea and hypoxemia. Her respiratory condition deteriorated enough to require introducing a ventilator on day 69 after HSCT. Chest computed tomography showed pleural effusion and diffuse ground-glass opacity with a centrilobular distribution. Bronchoalveolar lavage showed occult alveolar hemorrhage. Right cardiac catheterization confirmed the presence of pulmonary arterial hypertension and PVOD was suspected. Respiratory exacerbations resulted in death on day 157 after HSCT despite leukemia having been in remission. PVOD, though rare, should be considered in patients with unexplained respiratory impairment.