You Make the Call
Each month in “ You Make the Call ,” we pick a challenging clinical question submitted through the Consult a Colleague program and post the expert ’ s response , but we also want to know what you would do . Send in your response to next month ’ s clinical dilemma and see how your answer matches up to the expert ’ s in the next print issue .
This month , Swee Lay Thein , MD , discusses the pros and cons of splenectomy for a patient with hemoglobin H disease .
TRAINING and EDUCATION
Clinical Dilemma :
For many years I have been caring for a now 35-year-old woman with hemoglobin ( Hb ) H disease . Her long-term numbers are average ( Hb about 7.5 g / dL , reticulocytes about 7 %, bilirubin about 2.5 mg / dl ). She does not need transfusions , but accumulates iron and needs long-term deferasirox to prevent her ferritin from rising above 1,000 μg / L ( it was 1,700 in the past ). Would a splenectomy be a good option to improve her Hb , reduce iron overload , and improve quality of life ?
Expert Opinion
Swee Lay Thein , MD Senior Investigator Chief , Sickle Cell Branch National Heart , Lung and Blood Institute National Institutes of Health
Hereditary hemolytic anemias encompass a group of anemias with causes that affect integrity of the red cell membrane , involve red blood cell enzymes and hemoglobin ( hemoglobin variants and thalassemias ), and can include congenital dyserythropoietic anemias . A typical feature of these anemias is splenomegaly that can range from being just palpable to massive , causing immense abdominal discomfort with the possibility of splenic rupture . Due to concerns regarding short- and long-term complications , such as infections and thromboses , we tend not to recommend splenectomy for hereditary hemolysis . Apart for hereditary spherocytosis , the efficacy of splenectomy for the other anemias is not clear .
HbH disease is a clinically distinct form of the so-called “ non-transfusion-dependent thalassemias ” ( NTDTs ), a term used to label patients with hemoglobinopathies who do not require regular transfusion for survival . HbH disease is the most severe non-fatal form of α-thalassemia , caused by reduced production of α-globin arising from defects in the α-globin genes . The imbalanced globin chain synthesis leads to an excess of β chains forming tetramers ( HbH ) that are unstable and causes a phenotype of mild-to-moderate chronic hemolytic anemia ( 7-10 gm / dL ) and splenomegaly . I presume the patient with HbH disease has a large spleen . Does she have evidence of hypersplenism ?
Splenectomy should be considered when there is worsening anemia , evidence of hypersplenism , abdominal discomfort , left upper abdominal quadrant pain and early satiety . Possible splenic rupture is also a concern when splenomegaly is massive ( reaching suprapubic level ). Due to possible post-splenectomy complications , splenectomy should be a last resort . If hypersplenism or abdominal discomfort is a problem , I would use hydroxyurea initially , starting with a low dose ( 500 mg / day ) and titrating upwards ( monitoring white cell , platelet , and reticulocyte counts ). Hydroxyurea can be very effective in reducing extramedullary hemopoiesis and the splenic size .
The iron overload arises from increased gastrointestinal iron absorption . Removing the spleen will not reduce her iron overload . If anything , the spleen may have a scavenging effect on toxic iron species . I would continue deferasirox and monitor the iron saturation ( a better measure of toxic iron ). A baseline MRI for liver iron and cardiac iron is recommended and I recommend once- or twice-yearly MRI depending on initial score with ferritin assays for monitoring iron loading in between .
REFERENCES
1 . Taher AT , Vichinsky E , Musallam KM , Cappellini MD , Viprakasit V eds . Guidelines for the management of non transfusion dependent thalassaemia ( NTDT ) ( ed First ). Nicosia , Cyprus : Thalassaemia International Federation ; 2013 .
2 . Porter JB , Cappellini MD , Kattamis A , et al . Iron overload across the spectrum of non-transfusion-dependent thalassaemias : role of erythropoiesis , splenectomy and transfusions . Br J Haematol . 2017 ; 176 ( 2 ): 288-299 .
3 . Iolascon A , Andolfo I , Barcellini W , et al . Recommendations regarding splenectomy in hereditary hemolytic anemias . Haematologica . 2017 ; 102 ( 8 ): 1304-1313 .
4 . Fucharoen S , Viprakasit V . Hb H disease : clinical course and disease modifiers . Hematology Am Soc Hematol Educ Program . 2009:26-34 .
Next Month ’ s Clinical Dilemma :
A 55-year-old female patient received a diagnosis of chronic-phase chronic myeloid leukemia in April 2016 . After treatment with dasatinib 100 mg daily , major molecular response was achieved in January 2017 . BCR / ABL1 b2a2 increased to 0.99 percent by June 2017 , and dasatinib was increased to 140 mg daily ( BCR / ABL1 kinase domain mutation was negative ). When BCR / ABL reached 0.27 percent in January 2018 , she was changed to nilotinib 300 mg twice a day . BCR / ABL was 0.24 percent in April 2018 , and 0.77 percent in June 2018 . She is healthy and compliant . What should be done next ?
How would you respond ? Email us at ashclinicalnews @ hematology . org . ●
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42 ASH Clinical News August 2018