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The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry

Authors :
Schwab, Marisa E.
Lianoglou, Billie R.
Gano, Dawn
Gonzalez Velez, Juan
Allen, Isabel E.
Arvon, Regina
Baschat, Ahmet
Bianchi, Diana W.
Bitanga, Melissa
Bourguignon, Anne
Brown, Richard N.
Chen, Bruce
Chien, May
Davis-Nelson, Shareece
de Laat, Monique W. M.
Ekwattanakit, Supachai
Gollin, Yvonne
Hirata, Greigh
Jelin, Angie
Jolley, Jennifer
Meyer, Paul
Miller, Jena
Norton, Mary E.
Ogasawara, Keith K.
Panchalee, Tachjaree
Schindewolf, Erica
Shaw, Steven W.
Stumbaugh, Tammy
Thompson, Alexis A.
Towner, Dena
Tsai, Pai-Jong Stacy
Viprakasit, Vip
Volanakis, Emmanuel
Zhang, Li
Vichinsky, Elliott
MacKenzie, Tippi C.
Source :
Blood Advances; January 2023, Vol. 7 Issue: 2 p269-279, 11p
Publication Year :
2023

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.

Details

Language :
English
ISSN :
24739529 and 24739537
Volume :
7
Issue :
2
Database :
Supplemental Index
Journal :
Blood Advances
Publication Type :
Periodical
Accession number :
ejs61704574
Full Text :
https://doi.org/10.1182/bloodadvances.2022007823