Current treatments for wAIHA
| Treatment . | Dose schedule . | Response rate, % . | Time to response . | Comments . | Side effects/cons . | Ref. . |
|---|---|---|---|---|---|---|
| Predniso(lo)ne | 1-2 mg/kg per day for 3-4 wk | 80-90 (estimated cure rate in 20-30 only) | 7-25 d | Gradual tapering during a period no shorter than 4-6 mo | Diabetes mellitus, hypertension, peptic ulcer, osteoporosis, adrenal suppression, myopathy, psychosis, delayed wound healing, insomnia, menstrual irregularity, weight gain | 9,14,16 |
| Steroid boluses may be used for acute severe forms (ie, methylprednisolone 250 mg IV daily for 3 d) | ||||||
| IVIg | 0.4 g/kg per day for 5 d | 30-40 | 1-5 d | Responses usually last for about 3 wk | Infusion reactions particularly in patients with IgA deficiency, thromboembolic events, acute renal failure, increased serum viscosity | 16,71 |
| Advised in addition to steroids in critically ill patients, particularly during severe infections/sepsis | ||||||
| Rituximab | 375 mg/m2 per week for 4 wk | ∼80 (relapse- free survival of ∼60 at 3 y) | 3-6 wk | Other schedules include: (a) low dose (100 mg weekly for 4 wk) in patients with nonsevere hemolytic anemia, and in the elderly | Infusion reactions, late-onset neutropenia, hypogammaglobulinemia, reactivation of underlying infections (HBV, HCV, HIV, tuberculosis, etc) | 10-12,18 |
| (b) 1 g days 1 and 15, particularly in wAIHA associated with other autoimmune diseases | Regarding HBV reactivation, lamivudine prophylaxis up to 18 mo is recommended for anti-HBc Ab and/or anti-HBs Ab+ patients (if not vaccinated) | |||||
| Splenectomy | n/a | ∼80 (curative rate 20-50) | 7-10 d | Discouraged for patients older than 65-70 y, with cardiopulmonary disorders, thrombotic risk, immunodeficiencies, lymphoproliferative diseases, and systemic autoimmune conditions | Possible complications include serious infections (vaccinations warranted against Neisseria meningitidis ACWY and B type, pneumococcal, and Haemophilus influenzae type b; annual flu vaccine; variable schedules for 5 yearly boosters) and thrombotic events | 16,20-23 |
| Azathioprine | 2-4 mg/kg per day | ∼60 (usually with steroids) | 1-3 mo | Advised as steroid-sparing agent in AIHAs secondary to systemic autoimmune conditions, inflammatory bowel diseases, and autoimmune hepatitis | Myelotoxicity, particularly in case of thiopurine methyltransferase deficiency (start with 50 mg daily, and increase up to 150 mg in the absence of neutropenia), liver toxicity | 16,27 |
| Cyclosporine | 2.5 mg/kg, twice per day | ∼60 | 1-3 mo | Advised as steroid-sparing agent, particularly in AIHAs secondary to autoimmune conditions, Evans syndrome, and in case of features of BMF | Kidney damage, hypertension, infections, nausea, excessive hair growth | 16,24 |
| Cyclo-phosphamide | 50-100 mg per day or 800 mg/m2 IV monthly for 4-5 cycles | 50-70 | 2-6 wk | May be considered in cases of highly hemolytic disease, particularly if secondary to connective tissue disorders and lymphoproliferative diseases | Myelosuppression, infections, urotoxicity, secondary malignancy, teratogenicity, infertility | 3,16,25 |
| Mycophenolate | 500 mg, twice per day | 25-100 (small case series) | 1-3 mo | Mainly used in the pediatric setting | Nausea, headache, diarrhea | 26 |
| Danazol | 200 mg, 3 times per day | 20-50 | 1-3 mo | Steroid-sparing properties | Androgenic effects (to be avoided in men with prostatic adenoma or carcinoma), liver toxicity | 60,61 |
| Treatment . | Dose schedule . | Response rate, % . | Time to response . | Comments . | Side effects/cons . | Ref. . |
|---|---|---|---|---|---|---|
| Predniso(lo)ne | 1-2 mg/kg per day for 3-4 wk | 80-90 (estimated cure rate in 20-30 only) | 7-25 d | Gradual tapering during a period no shorter than 4-6 mo | Diabetes mellitus, hypertension, peptic ulcer, osteoporosis, adrenal suppression, myopathy, psychosis, delayed wound healing, insomnia, menstrual irregularity, weight gain | 9,14,16 |
| Steroid boluses may be used for acute severe forms (ie, methylprednisolone 250 mg IV daily for 3 d) | ||||||
| IVIg | 0.4 g/kg per day for 5 d | 30-40 | 1-5 d | Responses usually last for about 3 wk | Infusion reactions particularly in patients with IgA deficiency, thromboembolic events, acute renal failure, increased serum viscosity | 16,71 |
| Advised in addition to steroids in critically ill patients, particularly during severe infections/sepsis | ||||||
| Rituximab | 375 mg/m2 per week for 4 wk | ∼80 (relapse- free survival of ∼60 at 3 y) | 3-6 wk | Other schedules include: (a) low dose (100 mg weekly for 4 wk) in patients with nonsevere hemolytic anemia, and in the elderly | Infusion reactions, late-onset neutropenia, hypogammaglobulinemia, reactivation of underlying infections (HBV, HCV, HIV, tuberculosis, etc) | 10-12,18 |
| (b) 1 g days 1 and 15, particularly in wAIHA associated with other autoimmune diseases | Regarding HBV reactivation, lamivudine prophylaxis up to 18 mo is recommended for anti-HBc Ab and/or anti-HBs Ab+ patients (if not vaccinated) | |||||
| Splenectomy | n/a | ∼80 (curative rate 20-50) | 7-10 d | Discouraged for patients older than 65-70 y, with cardiopulmonary disorders, thrombotic risk, immunodeficiencies, lymphoproliferative diseases, and systemic autoimmune conditions | Possible complications include serious infections (vaccinations warranted against Neisseria meningitidis ACWY and B type, pneumococcal, and Haemophilus influenzae type b; annual flu vaccine; variable schedules for 5 yearly boosters) and thrombotic events | 16,20-23 |
| Azathioprine | 2-4 mg/kg per day | ∼60 (usually with steroids) | 1-3 mo | Advised as steroid-sparing agent in AIHAs secondary to systemic autoimmune conditions, inflammatory bowel diseases, and autoimmune hepatitis | Myelotoxicity, particularly in case of thiopurine methyltransferase deficiency (start with 50 mg daily, and increase up to 150 mg in the absence of neutropenia), liver toxicity | 16,27 |
| Cyclosporine | 2.5 mg/kg, twice per day | ∼60 | 1-3 mo | Advised as steroid-sparing agent, particularly in AIHAs secondary to autoimmune conditions, Evans syndrome, and in case of features of BMF | Kidney damage, hypertension, infections, nausea, excessive hair growth | 16,24 |
| Cyclo-phosphamide | 50-100 mg per day or 800 mg/m2 IV monthly for 4-5 cycles | 50-70 | 2-6 wk | May be considered in cases of highly hemolytic disease, particularly if secondary to connective tissue disorders and lymphoproliferative diseases | Myelosuppression, infections, urotoxicity, secondary malignancy, teratogenicity, infertility | 3,16,25 |
| Mycophenolate | 500 mg, twice per day | 25-100 (small case series) | 1-3 mo | Mainly used in the pediatric setting | Nausea, headache, diarrhea | 26 |
| Danazol | 200 mg, 3 times per day | 20-50 | 1-3 mo | Steroid-sparing properties | Androgenic effects (to be avoided in men with prostatic adenoma or carcinoma), liver toxicity | 60,61 |
ACWY, serogroups A, C, W, and Y; n/a, not applicable; Ref., reference.