Table 2.

Response to Eculizumab Therapy in Patients with HSCT-TMA

	Responders				Non-responders
	Patient 1	Patient 2	Patient 3	Patient 4	Patient 5	Patient 6
Response to eculizumab
Therapeutic eculizumab level (trough) after first dose	No	No	Yes	Yes	No	No
Therapeutic eculizumab level (trough) after subsequent doses	Yes	Yes	Yes	Yes	No*	No*
Normalization of LDH, d	16	18	27	15	NR	NR
Normalization of haptoglobin, d	25	43	15	15	8	NR
Disappearance of schistocytes, d	30	45	14	7	NR	NR
Normalization of hemoglobin without transfusions, d	20	43	23	7	NR	NR
Recovery of platelets to 20,000 K/μL, d	18	43	14	7	NR	NR
Recovery of platelets to 50,000 K/μL, d	30	45	14	7	NR	NR
Doubling of cystatin C–eGFR, d	31	95	141	16	NR	NR
Urine protein-to-creatinine ratio <2 mg/mg, d	19	108	63	29	NR	NR
Number of anti-HTN medication at the start of eculizumab therapy	7	6	6	9	6	7
Number of anti-HTN medication at the end of eculizumab therapy	0	1	2	2	4	2
Hematologic HSCT-TMA response, d	30	45	27	15	NR	NR
Complete resolution of HSCT-TMA, d	31	108	141	29	NR	NR
Outcomes
Current status	Alive	Alive	Alive	Alive	Dead	Dead
Time from starting eculizumab therapy to the last encounter, weeks	38	55	40	72	5	<2
Most current cystatin C–eGFR, mL/min	138	48	41	150	n/a	n/a
Most current random urine protein-to-creatinine ratio (normal <.2 mg/mg, nephrotic range >2 mg/mg)	.2	.9	.48	.14	n/a	n/a

LDH, lactate dehydrogenase; HTN, hypertension; NR, no response.

^*Did not achieve therapeutic eculizumab level even with the dose escalation.