Clinical Study

The First Asian, Single-Center Experience of Blastocyst Preimplantation Genetic Diagnosis with HLA Matching in Thailand for the Prevention of Thalassemia and Subsequent Curative Hematopoietic Stem Cell Transplantation of Twelve Affected Siblings

Table 4

Details and outcomes of PGD-HLA HSCT.

Recipient sexPGD-HLA donor sex and DxaPatient originAffected sibling thalassemia mutation diagnosisHSC sourceRecipient age at HSCT (months)HSCT status/country HSCT performedHSCT-related complications

1MM (normal)ThailandBThal/HbE Cd.17 (A>T)/HbEUCB59Success/ThailandNone
2FF (HbE trait)ThailandBThal/HbE HbE/IVSII-654 (C>T)UCB45Success/ThailandNone
3FM (HbE trait)ThailandBThal/HbE Cd.41/42 (-TCTT)/HbEUCB67Success/ThailandNone
4MF (HbE trait)ThailandBThal/HbE Cd.41/42 (-TCTT)/HbEUCB39Success/ThailandNone
5MF/F (IVSI-5)ThailandBThal/HbE HbE/IVSI-5 (G>C)UCB twins56Success/ThailandMild VOD, completely resolved
6FF (normal)ThailandhomoB-thal IVSII-654 (C>T)/Cd.41/42 (-TCTT)UCB45Success ThailandNone
7MM (Beta-28)ThailandhomoB-thal beta-28 (A>G)/Cd.17 (A>T)UCB in/ex utero collection36Success/ThailandNone
8FF (HbE trait)ThailandB-thal/HbE HbE/IVSII-654 (C>T)UCB + BMc (BM donor age 76 months)202Success/ThailandGrade 2 acute GvHD currently regress on cyclosporine and prednisolone. Mild VOD, thrombocytopenia, and CMV reactivation, all completely resolved
9FM (normal)CambodiaB-thal/HbE HbE/Cd.17 (A>T)BMc (BM donor age 12 months) (UCB not used)132Success/IndiaNone
10MF (normal)CambodiaB-thal/HbE HbE/IVSII-654 (C>T)UCBAwaiting HSCT
11FM (normal)ChinahomoB-thal beta-28/Cd.41/42 (-TCTT)UCB + BMc (BM donor age 36 months)108Success/ChinaNone
12FM (normal)IndiahomoB-thal IVSI-5 (G>C)/IVSI-1 (G>A)UCB60Success/IndiaNone
13MM (carrier)IndiahomoB-thal IVSI-5 (G>C)/IVSI-5 (G>C)N/AbUnknown
14FM (IVSI-5)IndiahomoB-thal Cd.41/42 (TCTT)/IVSI-5 (G>C)UCB + BMc (BM donor age at 29 months)75Success/ThailandNone
15FF (αα/--SEA)VietnamHbH-CS disease (--/αCsα)UCBAwaiting HSCT

aDx = diagnosis. bN/A = not applicable. cSame donor. CMV = cytomegalovirus; VOD = veno-occlusive disease; GvHD = graft-versus-host disease.