The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry
Issued Date
2023-01-24
Resource Type
ISSN
24739529
eISSN
24739537
Scopus ID
2-s2.0-85149128072
Pubmed ID
36306387
Journal Title
Blood Advances
Volume
7
Issue
2
Start Page
269
End Page
279
Rights Holder(s)
SCOPUS
Bibliographic Citation
Blood Advances Vol.7 No.2 (2023) , 269-279
Suggested Citation
Schwab M.E., Lianoglou B.R., Gano D., Velez J.G., Allen I.E., Arvon R., Baschat A., Bianchi D.W., Bitanga M., Bourguignon A., Brown R.N., Chen B., Chien M., Davis-Nelson S., de Laat M.W.M., Ekwattanakit S., Gollin Y., Hirata G., Jelin A., Jolley J., Meyer P., Miller J., Norton M.E., Ogasawara K.K., Panchalee T., Schindewolf E., Shaw S.W., Stumbaugh T., Thompson A.A., Towner D., Stacy Tsai P.J., Viprakasit V., Volanakis E., Zhang L., Vichinsky E., MacKenzie T.C. The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry. Blood Advances Vol.7 No.2 (2023) , 269-279. 279. doi:10.1182/bloodadvances.2022007823 Retrieved from: https://repository.li.mahidol.ac.th/handle/20.500.14594/82453
Title
The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry
Author(s)
Schwab M.E.
Lianoglou B.R.
Gano D.
Velez J.G.
Allen I.E.
Arvon R.
Baschat A.
Bianchi D.W.
Bitanga M.
Bourguignon A.
Brown R.N.
Chen B.
Chien M.
Davis-Nelson S.
de Laat M.W.M.
Ekwattanakit S.
Gollin Y.
Hirata G.
Jelin A.
Jolley J.
Meyer P.
Miller J.
Norton M.E.
Ogasawara K.K.
Panchalee T.
Schindewolf E.
Shaw S.W.
Stumbaugh T.
Thompson A.A.
Towner D.
Stacy Tsai P.J.
Viprakasit V.
Volanakis E.
Zhang L.
Vichinsky E.
MacKenzie T.C.
Lianoglou B.R.
Gano D.
Velez J.G.
Allen I.E.
Arvon R.
Baschat A.
Bianchi D.W.
Bitanga M.
Bourguignon A.
Brown R.N.
Chen B.
Chien M.
Davis-Nelson S.
de Laat M.W.M.
Ekwattanakit S.
Gollin Y.
Hirata G.
Jelin A.
Jolley J.
Meyer P.
Miller J.
Norton M.E.
Ogasawara K.K.
Panchalee T.
Schindewolf E.
Shaw S.W.
Stumbaugh T.
Thompson A.A.
Towner D.
Stacy Tsai P.J.
Viprakasit V.
Volanakis E.
Zhang L.
Vichinsky E.
MacKenzie T.C.
Author's Affiliation
Siriraj Hospital
Loma Linda University
UCSF Benioff Children‘s Hospital
Stanford University School of Medicine
UCSF School of Medicine
Chang Gung Memorial Hospital
Vanderbilt University Medical Center
The Children's Hospital of Philadelphia
University of California, San Francisco
California Pacific Medical Center
Kaiser Permanente
Northwestern University Feinberg School of Medicine
Auckland City Hospital
John A. Burns School of Medicine
University of California, Irvine
National Institutes of Health (NIH)
Centre Universitaire de Santé McGill
Johns Hopkins University
Fetal Diagnostic Institute of the Pacific
Loma Linda University
UCSF Benioff Children‘s Hospital
Stanford University School of Medicine
UCSF School of Medicine
Chang Gung Memorial Hospital
Vanderbilt University Medical Center
The Children's Hospital of Philadelphia
University of California, San Francisco
California Pacific Medical Center
Kaiser Permanente
Northwestern University Feinberg School of Medicine
Auckland City Hospital
John A. Burns School of Medicine
University of California, Irvine
National Institutes of Health (NIH)
Centre Universitaire de Santé McGill
Johns Hopkins University
Fetal Diagnostic Institute of the Pacific
Other Contributor(s)
Abstract
Alpha thalassemia major (ATM) is a hemoglobinopathy that usually results in perinatal demise if in utero transfusions (IUTs) are not performed. We established an international registry (NCT04872179) to evaluate the impact of IUTs on survival to discharge (primary outcome) as well as perinatal and neurodevelopmental secondary outcomes. Forty-nine patients were diagnosed prenatally, 11 were diagnosed postnatally, and all 11 spontaneous survivor genotypes had preserved embryonic zeta-globin levels. We compared 3 groups of patients; group 1, prenatally diagnosed and alive at hospital discharge (n = 14), group 2, prenatally diagnosed and deceased perinatally (n = 5), and group 3, postnatally diagnosed and alive at hospital discharge (n = 11). Group 1 had better outcomes than groups 2 and 3 in terms of the resolution of hydrops, delivery closer to term, shorter hospitalizations, and more frequent average or greater neurodevelopmental outcomes. Earlier IUT initiation was correlated with higher neurodevelopmental (Vineland-3) scores (r = -0.72, P = .02). Preterm delivery after IUT was seen in 3/16 (19%) patients who continued their pregnancy. When we combined our data with those from 2 published series, patients who received ≥2 IUTs had better outcomes than those with 0 to 1 IUT, including resolution of hydrops, delivery at ≥34 weeks gestation, and 5-minute appearance, pulse, grimace, activity, and respiration scores ≥7. Neurodevelopmental assessments were normal in 17/18 of the ≥2 IUT vs 5/13 of the 0 to 1 IUT group (OR 2.74; P = .01). Thus, fetal transfusions enable the survival of patients with ATM and normal neurodevelopment, even in those patients presenting with hydrops. Nondirective prenatal counseling for expectant parents should include the option of IUTs.
