FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2912754533> ?p ?o ?g. }

• W2912754533 abstract "Introduction Thalassemia major is curable by allogeneic hematopoetic stem cell transplant (HSCT). Due to lack of matched unrelated donors most children of ethnic minorities are unable to access HSCT. Recent reports of haploidentical donor HSCT with reduced intensity conditioning (RIC) with post transplant cyclophosphamide (PTCy) from family donors in hemoglobinopathies have shown encouraging results. However, rejection remains a problem in children with thalassemia undergoing RIC haploidentical HSCT. Another need is to avoid total body irradiation (TBI) during conditioning in infants due to its effects on the developing brain. Traditionally for haploidentical HSCT with PTCy, Tacrolimus and Myecophenolate Mofetil (MMF) have been used as part of graft vs. host disease (GVHD) prophylaxis. However, intravenous tacrolimus is not easily available so in very young children difficult to achieve good levels with oral tacrolimus which can increase risk of rejection. Here we describe successful haploidentical HSCT in an infant with myeloablative conditioning (free of radiotherapy) and PTCy with intravenous cyclosporine and oral MMF as GVHD prophylaxis. Methods A 10-month-old, male infant with thalassemia major (Pesaro class I) who had no matched related or unrelated donor available underwent haploidentical HSCT after taking informed consent of the parents in December 2017. Donor was his mother who was 5/10 matched. He had been transfused just twice before HSCT. The conditioning was with Rituximab 100 mg/m2 IV on day -11, rabbit anti-thymoglubulin (thymoglobulin) 1.5 mg/kg/dose daily for 3 days (Day-10 to -8) Thiotepa 10 mg/kg/dose intravenous (IV) for 1 day (Day-7), Busulfan 4 mg/kg/dose daily IV for 4 days (Day -6 to -3) and Fludarabine 40 mg/m2/dose daily IV for 4 days (Day -6 to -3). Peripheral blood stem cells (10 million/kg CD34+ cells) were harvested from mother and transfused to the patient on Day 0. GVHD prophylaxis was with PTCy 50 mg/kg on Day+3 & 4, intravenous cyclosporine from day+5 (targeting levels 150-250 ng/ml) and MMF from day+5. Results His neutrophils engrafted on day+14 and platelets on day+16. He developed high grade fever from day+2 till day+5 which disappeared after PTCy. Chimerism on day+30, 100 and 12 months was fully donor. He developed no acute or chronic GVHD. At 6 months his lymphocyte counts showed CD4-400/ul, CD8-840/ul, CD19-8/ul and CD16/56-120/ul. His viral studies by PCR for cytomegalovirus, BK virus and Ebstein-Barr virus remained negative throughout. MMF was stopped on Day+35.He is disease free and GVHD free on day+400 post HSCT and is off cyclosporine. Conclusion Busulfan, Fludarabine and Thiotepa based meyeloablative conditioning is a radiotherapy free option for infants undergoing haploidentical HSCT with PTCy for thalassemia major. For GVHD prophylaxis in haploidentical HSCT with PTCy intravenous cyclosporine can be an alternative to oral tacrolimus." @default.
• W2912754533 created "2019-02-21" @default.
• W2912754533 creator A5005203511 @default.
• W2912754533 creator A5025478187 @default.
• W2912754533 creator A5032854729 @default.
• W2912754533 creator A5079070873 @default.
• W2912754533 creator A5090931617 @default.
• W2912754533 date "2019-03-01" @default.
• W2912754533 modified "2023-09-23" @default.
• W2912754533 title "Successful Haploidentical Stem Cell Transplant with Busulfan, Fludarabine, Thiotepa Conditioning and Post Transplant Cyclophosphamide, Cyclosporine and Mycophenolate Mofetil in an Infant with Thalassemia Major" @default.
• W2912754533 doi "https://doi.org/10.1016/j.bbmt.2018.12.647" @default.
• W2912754533 hasPublicationYear "2019" @default.
• W2912754533 type Work @default.
• W2912754533 sameAs 2912754533 @default.
• W2912754533 citedByCount "0" @default.
• W2912754533 crossrefType "journal-article" @default.
• W2912754533 hasAuthorship W2912754533A5005203511 @default.
• W2912754533 hasAuthorship W2912754533A5025478187 @default.
• W2912754533 hasAuthorship W2912754533A5032854729 @default.
• W2912754533 hasAuthorship W2912754533A5079070873 @default.
• W2912754533 hasAuthorship W2912754533A5090931617 @default.
• W2912754533 hasBestOaLocation W29127545331 @default.
• W2912754533 hasConcept C126322002 @default.
• W2912754533 hasConcept C141071460 @default.
• W2912754533 hasConcept C187212893 @default.
• W2912754533 hasConcept C2776694085 @default.
• W2912754533 hasConcept C2776755627 @default.
• W2912754533 hasConcept C2777408962 @default.
• W2912754533 hasConcept C2777630719 @default.
• W2912754533 hasConcept C2777799968 @default.
• W2912754533 hasConcept C2778880498 @default.
• W2912754533 hasConcept C2779263901 @default.
• W2912754533 hasConcept C2779338263 @default.
• W2912754533 hasConcept C2779968505 @default.
• W2912754533 hasConcept C2780611847 @default.
• W2912754533 hasConcept C2780653079 @default.
• W2912754533 hasConcept C2909675724 @default.
• W2912754533 hasConcept C2911091166 @default.
• W2912754533 hasConcept C71924100 @default.
• W2912754533 hasConceptScore W2912754533C126322002 @default.
• W2912754533 hasConceptScore W2912754533C141071460 @default.
• W2912754533 hasConceptScore W2912754533C187212893 @default.
• W2912754533 hasConceptScore W2912754533C2776694085 @default.
• W2912754533 hasConceptScore W2912754533C2776755627 @default.
• W2912754533 hasConceptScore W2912754533C2777408962 @default.
• W2912754533 hasConceptScore W2912754533C2777630719 @default.
• W2912754533 hasConceptScore W2912754533C2777799968 @default.
• W2912754533 hasConceptScore W2912754533C2778880498 @default.
• W2912754533 hasConceptScore W2912754533C2779263901 @default.
• W2912754533 hasConceptScore W2912754533C2779338263 @default.
• W2912754533 hasConceptScore W2912754533C2779968505 @default.
• W2912754533 hasConceptScore W2912754533C2780611847 @default.
• W2912754533 hasConceptScore W2912754533C2780653079 @default.
• W2912754533 hasConceptScore W2912754533C2909675724 @default.
• W2912754533 hasConceptScore W2912754533C2911091166 @default.
• W2912754533 hasConceptScore W2912754533C71924100 @default.
• W2912754533 hasLocation W29127545331 @default.
• W2912754533 hasOpenAccess W2912754533 @default.
• W2912754533 hasPrimaryLocation W29127545331 @default.
• W2912754533 hasRelatedWork W2489705409 @default.
• W2912754533 hasRelatedWork W2615902868 @default.
• W2912754533 hasRelatedWork W2884744511 @default.
• W2912754533 hasRelatedWork W2912754533 @default.
• W2912754533 hasRelatedWork W2945080012 @default.
• W2912754533 hasRelatedWork W2986047317 @default.
• W2912754533 hasRelatedWork W3001222592 @default.
• W2912754533 hasRelatedWork W3108266830 @default.
• W2912754533 hasRelatedWork W3216809241 @default.
• W2912754533 hasRelatedWork W4205680087 @default.
• W2912754533 isParatext "false" @default.
• W2912754533 isRetracted "false" @default.
• W2912754533 magId "2912754533" @default.
• W2912754533 workType "article" @default.