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Outcomes of allogeneic transplantation for hemoglobin Bart’s hydrops fetalis syndrome in Hong Kong
Author(s) -
Chan Wilson Y. K.,
Lee Pamela P. W.,
Lee Vincent,
Chan Godfrey C. F.,
Leung Wing,
Ha S. Y.,
Cheuk Daniel K. L.
Publication year - 2021
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.14037
Subject(s) - medicine , sibling , pediatrics , hydrops fetalis , transplantation , hematopoietic stem cell transplantation , cord blood , retrospective cohort study , surgery , pregnancy , fetus , psychology , developmental psychology , biology , genetics
Background Hemoglobin Bart's hydrops fetalis syndrome (BHFS) was once considered a fatal condition universally. Medical advances over the past three decades have resulted in increasing numbers of BHFS survivors. This retrospective review summarized local territory‐wide experience and outcomes of BHFS patients who received allogeneic hematopoietic stem cell transplantation (HSCT) in Hong Kong. Methods All BHFS patients who underwent allogeneic HSCT in Hong Kong, either in one of the two former pediatric transplant centers (Queen Mary Hospital and Prince of Wales Hospital) on or before 2019 or in the single territory‐wide pediatric transplant center (Hong Kong Children's Hospital) since 2019, from January 1, 1996, till December 31, 2020, were included. Basic demographic data, perinatal history, transplant details, long‐term outcomes, and morbidities were reviewed. Results Total five allogeneic HSCT were performed in two males and three females at a median age of 22 months, which include one 8/8 matched‐sibling bone marrow transplant, one 5/6 matched‐sibling cord blood transplant with HLA‐DR antigenic mismatch, two 12/12 matched‐unrelated peripheral blood stem cell transplant (PBSCT), and one haploidentical PBSCT with TCRαβ/CD45RA depletion from maternal donor. Neutrophil and platelet engrafted (>20 × 10 9 /L) at a median of 15 and 22 days, respectively. All achieved near full donor chimerism at 1 month. All patients survived and remained transfusion‐independent without significant morbidities with median follow‐up duration of 10 years. Conclusion To conclude, local data demonstrated favorable outcome of allogeneic HSCT for BHFS patients, but sample number is small. Non‐directive approach in counseling and international collaboration is recommended.