Patients with AIHA treated with AIHA-directed pharmacologic
therapy (Fig. 1)
Of the 33 patients with AIHA, only 16 (48.5%) were treated with new
AIHA-directed pharmacologic therapy. Patients received a median of one
AIHA-directed treatment (range: 1-4). Treatments included:
corticosteroids (n=13), rituximab (n=6), intravenous immunoglobulin
(n=2), mycophenolate (n=2), erythropoietin (n=2), bortezomib (n=2),
cyclosporine (n=1), and plasmapheresis (n=1). Variable treatment
responses were seen (Table 2). Six patients required corticosteroids
only, 1 patient received rituximab only, 2 patients received an
erythropoietin stimulating agent only, and 7 patients received more than
one AIHA-directed treatment. First-line pharmacologic treatment was most
commonly corticosteroids (n=11). The most common second line treatment
was rituximab (n=4).
Corticosteroid only treatment . Of the 6 patients who were treated
with corticosteroids only, the median time to development of AIHA after
HSCT was 5.9 months (range: 3.7-29.7 months). These patients were a
median age of 0.46 years (range: 0.14-3.88 years) at time of transplant.
Five of these patients had received bone marrow (BM), and 1 had received
peripheral blood stem cells (PBSC). All patients in this group had an
immunodeficiency diagnosis, including CD40L deficiency (n=2), severe
combined immunodeficiency (n=2), Wiskott-Aldrich Syndrome (n=1), and
NFKB1 deficiency (n=1). Five patients received serotherapy with
anti-thymocyte globulin and 5 received myeloablative conditioning. One
patient had CMV while 2 had EBV concurrently or prior to the time of
AIHA after transplant. At the time of AIHA diagnosis, these patients had
a median hemoglobin of 6.4 g/dl (range 4 -10 g/dL). All patients
received pRBC transfusions. Five patients (83.3%) had a partial
response to corticosteroids at 1 month; one patient had a complete
response. One of these patients was diagnosed with EBV 1 week after
being diagnosed with AIHA and received rituximab for treatment of EBV.
Five patients (83.3%) were in complete remission at last follow up
while 1 patient had a partial remission.
Corticosteroids and rituximab treatment . Three patients received
corticosteroids followed by rituximab for treatment of AIHA. The median
time to AIHA after HSCT in these patients was 6.34 months (range
4.17-6.77 months) and were a median age of 4.98 years (range 1.29-8.04
years) at the time of transplant. Median hemoglobin was 4.8 g/dL (range
4.0-6.9 g/dL). These patients all had non-malignant diagnoses and
received matched unrelated donor (MURD) BM transplants with
myeloablative conditioning. One month after rituximab, 2 patients had a
partial response and 1 patient had a complete response. Six months after
AIHA diagnosis, 1 patient was in partial remission and 2 patients were
in complete remission; all were in complete remission at last follow up.
Bortezomib treatment . Two patients required third- or fourth-line
treatment with bortezomib. One patient developed AIHA at 4.73 months
after MURD BM transplant with reduced intensity conditioning including
alemtuzumab serotherapy for X-linked lymphoproliferative disease.
Hemoglobin at time of diagnosis was 4 g/dL, and the patient received 10
pRBC transfusions in the first two weeks after diagnosis and required
ICU care. The patient was started initially on corticosteroids, then
rituximab, followed by mycophenolate with non-response and then received
bortezomib with a partial response. The patient developed iron overload
the year following AIHA diagnosed by T2* MRI. At the time of last follow
up, the patient was alive and in complete remission.
The other patient developed AIHA at 3.19 months post-transplant after a
matched related BM transplant with myeloablative conditioning for
combined immunodeficiency. This patient had a history of AIHA
pre-transplant. Hemoglobin at time of AIHA diagnosis was 7.1 g/dL, and
the patient received 2 pRBC transfusions in the first two weeks after
diagnosis. The patient received plasmapheresis after intravenous
immunoglobulin with non-response, corticosteroids with partial response,
and then bortezomib with non-response. This patient also had iron
overload in the year following AIHA diagnosed by T2* MRI. This patient
later developed pure red cell aplasia and graft failure and died after
receiving a second transplant.