{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,28]],"date-time":"2026-03-28T20:50:16Z","timestamp":1774731016670,"version":"3.50.1"},"reference-count":20,"publisher":"Springer Science and Business Media LLC","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,3,28]]},"DOI":"10.7759\/cureus.106037","type":"journal-article","created":{"date-parts":[[2026,3,28]],"date-time":"2026-03-28T20:15:41Z","timestamp":1774728941000},"source":"Crossref","is-referenced-by-count":0,"title":["Navigating Haploidentical Stem Cell Transplantation in Congenital Amegakaryocytic Thrombocytopenia With Prior Intracranial Bleeding: Report of Two Pediatric Cases"],"prefix":"10.7759","author":[{"given":"Aditi","family":"Tulsiyan","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nita","family":"Radhakrishnan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anuj","family":"Singh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sudipto","family":"Bhattacharya","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hari","family":"Gaire","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Satyam","family":"Arora","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seema","family":"Dua","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Savitri","family":"Singh","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anukriti","family":"Srivastava","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","reference":[{"key":"ref1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.v97.1.139","article-title":"c-mpl mutations are the cause of congenital amegakaryocytic thrombocytopenia","volume":"97","author":"Ballmaier M","year":"2001","unstructured":"Ballmaier M, Germeshausen M, Schulze H, et al.. c-mpl mutations are the cause of congenital amegakaryocytic thrombocytopenia. Blood. 2001, 97:139-46. 10.1182\/blood.v97.1.139","journal-title":"Blood"},{"key":"ref2","doi-asserted-by":"publisher","DOI":"10.1016\/j.beha.2021.101286","article-title":"Congenital amegakaryocytic thrombocytopenia - Not a single disease","volume":"34","author":"Germeshausen M","year":"2021","unstructured":"Germeshausen M, Ballmaier M. Congenital amegakaryocytic thrombocytopenia - Not a single disease. Best Pract Res Clin Haematol. 2021, 34:101286. 10.1016\/j.beha.2021.101286","journal-title":"Best Pract Res Clin Haematol"},{"key":"ref3","doi-asserted-by":"publisher","DOI":"10.1126\/science.8073287","article-title":"Thrombocytopenia in c-mpl-deficient mice","volume":"265","author":"Gurney AL","year":"1994","unstructured":"Gurney AL, Carver-Moore K, de Sauvage FJ, Moore MW. Thrombocytopenia in c-mpl-deficient mice. Science. 1994, 265:1445-7. 10.1126\/science.8073287","journal-title":"Science"},{"key":"ref4","doi-asserted-by":"publisher","DOI":"10.1182\/asheducation-2017.1.88","article-title":"Inherited bone marrow failure syndromes: considerations pre- and posttransplant","volume":"2017","author":"Alter BP","year":"2017","unstructured":"Alter BP. Inherited bone marrow failure syndromes: considerations pre- and posttransplant. Hematology Am Soc Hematol Educ Program. 2017, 2017:88-95. 10.1182\/asheducation-2017.1.88","journal-title":"Hematology Am Soc Hematol Educ Program"},{"key":"ref5","doi-asserted-by":"publisher","DOI":"10.1002\/pbc.22927","article-title":"Congenital amegakaryocytic thrombocytopenia","volume":"57","author":"Geddis AE","year":"2011","unstructured":"Geddis AE. Congenital amegakaryocytic thrombocytopenia. Pediatr Blood Cancer. 2011, 57:199-203. 10.1002\/pbc.22927","journal-title":"Pediatr Blood Cancer"},{"key":"ref6","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2020.257972","article-title":"CAMT-MPL: congenital amegakaryocytic thrombocytopenia caused by MPL mutations - heterogeneity of a monogenic disorder - a comprehensive analysis of 56 patients","volume":"106","author":"Germeshausen M","year":"2021","unstructured":"Germeshausen M, Ballmaier M. CAMT-MPL: congenital amegakaryocytic thrombocytopenia caused by MPL mutations - heterogeneity of a monogenic disorder - a comprehensive analysis of 56 patients. Haematologica. 2021, 106:2439-48. 10.3324\/haematol.2020.257972","journal-title":"Haematologica"},{"key":"ref7","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2017.178111","article-title":"Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up","volume":"103","author":"Alter BP","year":"2018","unstructured":"Alter BP, Giri N, Savage SA, Rosenberg PS. Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up. Haematologica. 2018, 103:30-9. 10.3324\/haematol.2017.178111","journal-title":"Haematologica"},{"key":"ref8","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-023-05347-7","article-title":"Genomic and computational analysis of four novel variants of MPL gene in congenital amegakaryocytic thrombocytopenia","volume":"102","author":"Shah A","year":"2023","unstructured":"Shah A, Kumar C, Shanmukhaiah C, Rajendran A, Mudaliar S, Idicula-Thomas S, Vundinti BR. Genomic and computational analysis of four novel variants of MPL gene in congenital amegakaryocytic thrombocytopenia. Ann Hematol. 2023, 102:2683-93. 10.1007\/s00277-023-05347-7","journal-title":"Ann Hematol"},{"key":"ref9","article-title":"Congenital amegakaryocytic thrombocytopenia (CAMT): a case report with review of literature","volume":"50","author":"Desai SR","year":"2007","unstructured":"Desai SR, Ranade SR. Congenital amegakaryocytic thrombocytopenia (CAMT): a case report with review of literature. Indian J Pathol Microbiol. 2007, 50:659-60.","journal-title":"Indian J Pathol Microbiol"},{"key":"ref10","doi-asserted-by":"publisher","DOI":"10.1111\/petr.12175","article-title":"Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: report of a case and review of the literature","volume":"18","author":"Woods G","year":"2014","unstructured":"Woods G, Bajwa RP, Rose MJ. Reduced intensity transplantation for congenital amegakaryocytic thrombocytopenia: report of a case and review of the literature. Pediatr Transplant. 2014, 18:E31-4. 10.1111\/petr.12175","journal-title":"Pediatr Transplant"},{"key":"ref11","doi-asserted-by":"publisher","DOI":"10.1016\/j.jtct.2021.10.009","article-title":"Outcomes in hematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia","volume":"28","author":"Cancio M","year":"2022","unstructured":"Cancio M, Hebert K, Kim S, et al.. Outcomes in hematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia. Transplant Cell Ther. 2022, 28:101.e1-6. 10.1016\/j.jtct.2021.10.009","journal-title":"Transplant Cell Ther"},{"key":"ref12","doi-asserted-by":"publisher","DOI":"10.1038\/s41409-024-02416-x","article-title":"Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT","volume":"59","author":"Aldebert C","year":"2024","unstructured":"Aldebert C, Fahd M, Galimard JE, et al.. Outcomes of patients undergoing allogeneic haematopoietic stem cell transplantation for congenital amegakaryocytic thrombocytopenia; a study on behalf of the PDWP of the EBMT. Bone Marrow Transplant. 2024, 59:1717-25. 10.1038\/s41409-024-02416-x","journal-title":"Bone Marrow Transplant"},{"key":"ref13","doi-asserted-by":"publisher","DOI":"10.3389\/fped.2022.903872","article-title":"Non-myeloablative conditioning is sufficient to achieve complete donor myeloid chimerism following matched sibling donor bone marrow transplant for myeloproliferative leukemia virus oncogene (MPL) mutation-driven congenital amegakaryocytic thrombocytopenia: Case report","volume":"10","author":"Oved JH","year":"2022","unstructured":"Oved JH, Shah YB, Venella K, Paessler ME, Olson TS. Non-myeloablative conditioning is sufficient to achieve complete donor myeloid chimerism following matched sibling donor bone marrow transplant for myeloproliferative leukemia virus oncogene (MPL) mutation-driven congenital amegakaryocytic thrombocytopenia: Case report. Front Pediatr. 2022, 10:903872. 10.3389\/fped.2022.903872","journal-title":"Front Pediatr"},{"key":"ref14","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2141.2000.02099.x","article-title":"Haematopoietic stem cell transplantation for amegakaryocytic thrombocytopenia","volume":"109","author":"Lackner A","year":"2000","unstructured":"Lackner A, Basu O, Bierings M, et al.. Haematopoietic stem cell transplantation for amegakaryocytic thrombocytopenia. Br J Haematol. 2000, 109:773-5. 10.1046\/j.1365-2141.2000.02099.x","journal-title":"Br J Haematol"},{"key":"ref15","article-title":"Bone marrow transplantation in two children with congenital amegakaryocytic thrombocytopenia","volume":"15","author":"Henter JI","year":"1995","unstructured":"Henter JI, Winiarski J, Ljungman P, Ringd\u00e9n O, Ost A. Bone marrow transplantation in two children with congenital amegakaryocytic thrombocytopenia. Bone Marrow Transplant. 1995, 15:799-801.","journal-title":"Bone Marrow Transplant"},{"key":"ref16","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2019.03.009","article-title":"Haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for Primary Immunodeficiencies and inherited disorders in children","volume":"25","author":"Neven B","year":"2019","unstructured":"Neven B, Diana JS, Castelle M, et al.. Haploidentical hematopoietic stem cell transplantation with post-transplant cyclophosphamide for Primary Immunodeficiencies and inherited disorders in children. Biol Blood Marrow Transplant. 2019, 25:1363-73. 10.1016\/j.bbmt.2019.03.009","journal-title":"Biol Blood Marrow Transplant"},{"key":"ref17","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-03-563817","article-title":"HLA-haploidentical stem cell transplantation after removal of \u03b1\u03b2+ T and B cells in children with nonmalignant disorders","volume":"124","author":"Bertaina A","year":"2014","unstructured":"Bertaina A, Merli P, Rutella S, et al.. HLA-haploidentical stem cell transplantation after removal of \u03b1\u03b2+ T and B cells in children with nonmalignant disorders. Blood. 2014, 124:822-6. 10.1182\/blood-2014-03-563817","journal-title":"Blood"},{"key":"ref18","doi-asserted-by":"publisher","DOI":"10.1097\/MPH.0000000000000247","article-title":"Congenital amegakaryocytic thrombocytopenia: a case report of pediatric twins undergoing matched unrelated bone marrow transplantation","volume":"37","author":"Rao AA","year":"2015","unstructured":"Rao AA, Gourde JA, Marri P, Galardy PJ, Khan SP, Rodriguez V. Congenital amegakaryocytic thrombocytopenia: a case report of pediatric twins undergoing matched unrelated bone marrow transplantation. J Pediatr Hematol Oncol. 2015, 37:304-6. 10.1097\/MPH.0000000000000247","journal-title":"J Pediatr Hematol Oncol"},{"key":"ref19","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1702558","article-title":"Peripheral stem cell transplantation in a child with amegakaryocytic thrombocytopenia","volume":"26","author":"Ye\u015filipek","year":"2000","unstructured":"Ye\u015filipek, Hazar V, K\u00fcpesiz A, Yegin O. Peripheral stem cell transplantation in a child with amegakaryocytic thrombocytopenia. Bone Marrow Transplant. 2000, 26:571-2. 10.1038\/sj.bmt.1702558","journal-title":"Bone Marrow Transplant"},{"key":"ref20","doi-asserted-by":"publisher","DOI":"10.1016\/j.beha.2024.101556","article-title":"The role of registries in hematological disorders","volume":"37","author":"Baldomero H","year":"2024","unstructured":"Baldomero H, Neumann D, Hamad N, et al.. The role of registries in hematological disorders. Best Pract Res Clin Haematol. 2024, 37:101556. 10.1016\/j.beha.2024.101556","journal-title":"Best Pract Res Clin Haematol"}],"container-title":["Cureus"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.cureus.com\/articles\/467103-navigating-haploidentical-stem-cell-transplantation-in-congenital-amegakaryocytic-thrombocytopenia-with-prior-intracranial-bleeding-report-of-two-pediatric-cases","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,28]],"date-time":"2026-03-28T20:15:42Z","timestamp":1774728942000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.cureus.com\/articles\/467103-navigating-haploidentical-stem-cell-transplantation-in-congenital-amegakaryocytic-thrombocytopenia-with-prior-intracranial-bleeding-report-of-two-pediatric-cases"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,28]]},"references-count":20,"URL":"https:\/\/doi.org\/10.7759\/cureus.106037","relation":{},"ISSN":["2168-8184"],"issn-type":[{"value":"2168-8184","type":"print"}],"subject":[],"published":{"date-parts":[[2026,3,28]]}}}