{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T21:27:18Z","timestamp":1792272438029,"version":"4.4.0"},"reference-count":70,"publisher":"American Society of Hematology","issue":"11","content-domain":{"domain":["ashpublications.org"],"crossmark-restriction":true},"short-container-title":[],"published-print":{"date-parts":[[2020,9,10]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Pyruvate kinase deficiency (PKD) is an autosomal-recessive enzyme defect of the glycolytic pathway that causes congenital nonspherocytic hemolytic anemia. The diagnosis and management of patients with PKD can be challenging due to difficulties in the diagnostic evaluation and the heterogeneity of clinical manifestations, ranging from fetal hydrops and symptomatic anemia requiring lifelong transfusions to fully compensated hemolysis. Current treatment approaches are supportive and include transfusions, splenectomy, and chelation. Complications, including iron overload, bilirubin gallstones, extramedullary hematopoiesis, pulmonary hypertension, and thrombosis, are related to the chronic hemolytic anemia and its current management and can occur at any age. Disease-modifying therapies in clinical development may decrease symptoms and findings associated with chronic hemolysis and avoid the complications associated with current treatment approaches. As these disease-directed therapies are approved for clinical use, clinicians will need to define the types of symptoms and findings that determine the optimal patients and timing for initiating these therapies. In this article, we highlight disease manifestations, monitoring approaches, strategies for managing complications, and novel therapies in development.<\/jats:p>","DOI":"10.1182\/blood.2019000945","type":"journal-article","created":{"date-parts":[[2020,7,23]],"date-time":"2020-07-23T21:57:31Z","timestamp":1595541451000},"page":"1241-1249","update-policy":"https:\/\/doi.org\/10.1182\/blood.2019cm0000","source":"Crossref","is-referenced-by-count":75,"title":["Management of pyruvate kinase deficiency in children and adults"],"prefix":"10.1182","volume":"136","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7302-0449","authenticated-orcid":false,"given":"Rachael F.","family":"Grace","sequence":"first","affiliation":[{"name":"Dana-Farber\/Boston Children\u2019s Cancer and Blood Disorders Center, Boston, MA; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wilma","family":"Barcellini","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Ca\u2019 Granda Ospedale Maggiore Policlinico, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"234","reference":[{"issue":"5","key":"2020091011283882900_B1","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1182\/blood.V9.5.414.414","article-title":"Autohemolysis and other changes resulting from the incubation in vitro of red cells from patients with congenital hemolytic anemia","volume":"9","author":"Selwyn","year":"1954","journal-title":"Blood"},{"issue":"4","key":"2020091011283882900_B2","doi-asserted-by":"crossref","first-page":"1371","DOI":"10.1182\/blood.V16.4.1371.1371","article-title":"Nonspherocytic congenital hemolytic anemia","volume":"16","author":"De Gruchy","year":"1960","journal-title":"Blood"},{"key":"2020091011283882900_B3","first-page":"100","article-title":"A specific erythrocyte glycolytic enzyme defect (pyruvate kinase) in three subjects with congenital non-spherocytic hemolytic anemia","volume":"74","author":"Valentine","year":"1961","journal-title":"Trans Assoc Am Physicians"},{"issue":"3","key":"2020091011283882900_B4","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1182\/blood.V19.3.267.267","article-title":"Pyruvate kinase (PK) deficiency hereditary nonspherocytic hemolytic anemia","volume":"19","author":"Tanaka","year":"1962","journal-title":"Blood"},{"issue":"20","key":"2020091011283882900_B5","doi-asserted-by":"crossref","first-page":"1023","DOI":"10.1056\/NEJM196405142702001","article-title":"Extreme hemolysis and red-cell distortion in erythrocyte pyruvate kinase deficiency. 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