Table 3 Summary of selected... | American Society of Hematology

Table 3

Summary of selected interventions attempted and outcome for each of the children within the cohort

Case	Interventions			Outcome
Case	Regular transfusions	IVIg replacement	BMT	Alive/Dead	Age at death	Terminal event/Cause of death
1	Yes	Yes	No	Dead	14 y	Sepsis with multiorgan failure and toxic epidermal necrolysis (attributed to a cephalosporin)
2	No	Yes	No (awaited)	Alive (7 y)	n/a	n/a
3	Yes	Yes	No	Dead	61 mo	Multiorgan failure
4	Yes	Yes	No	Dead	4 y	Admission with seizures, hypokalemia and fevers; rapid onset multiorgan failure; adrenal hemorrhage on autopsy
5	Yes	?	No	Dead	56 mo	Arrived in shock with severe hypoglycemia; died within hours in multiorgan failure
6	Yes	Yes	No	Dead	25 mo	Cardiac failure secondary to cardiomyopathy (not related to iron overload)
7	Yes	Yes	No	Dead	16 mo	Multiorgan failure (pneumonitis; cardiac failure)
8	Yes	Yes	No	Alive (19 y)	n/a	n/a
9	No	No	No	Alive (5 y)	n/a	n/a
10	Yes	Yes	No	Dead	28 mo	Cardiac failure secondary to cardiomyopathy (not related to iron overload)
11	Yes*	Yes*	Yes (aged 9 mo)	Alive (4 y)	n/a	n/a
12	Yes	No	No	Alive (45 mo)	n/a	n/a

Case	Interventions			Outcome
Case	Regular transfusions	IVIg replacement	BMT	Alive/Dead	Age at death	Terminal event/Cause of death
1	Yes	Yes	No	Dead	14 y	Sepsis with multiorgan failure and toxic epidermal necrolysis (attributed to a cephalosporin)
2	No	Yes	No (awaited)	Alive (7 y)	n/a	n/a
3	Yes	Yes	No	Dead	61 mo	Multiorgan failure
4	Yes	Yes	No	Dead	4 y	Admission with seizures, hypokalemia and fevers; rapid onset multiorgan failure; adrenal hemorrhage on autopsy
5	Yes	?	No	Dead	56 mo	Arrived in shock with severe hypoglycemia; died within hours in multiorgan failure
6	Yes	Yes	No	Dead	25 mo	Cardiac failure secondary to cardiomyopathy (not related to iron overload)
7	Yes	Yes	No	Dead	16 mo	Multiorgan failure (pneumonitis; cardiac failure)
8	Yes	Yes	No	Alive (19 y)	n/a	n/a
9	No	No	No	Alive (5 y)	n/a	n/a
10	Yes	Yes	No	Dead	28 mo	Cardiac failure secondary to cardiomyopathy (not related to iron overload)
11	Yes*	Yes*	Yes (aged 9 mo)	Alive (4 y)	n/a	n/a
12	Yes	No	No	Alive (45 mo)	n/a	n/a

n/a, not applicable.

Child 11’s transfusion dependence and immunodeficiency fully resolved after BMT.