What are the treatment options for blood disorders in hematology?

Treatment Options for Blood Disorders in Hematology

Treatment for blood disorders must be selected based on the specific diagnosis, with iron deficiency anemia treated with oral ferrous sulfate, immune thrombocytopenia managed with corticosteroids followed by thrombopoietin receptor agonists or splenectomy, hemochromatosis treated with therapeutic phlebotomy, and hematologic malignancies requiring disease-specific chemotherapy, targeted agents, or stem cell transplantation.

Iron Deficiency Anemia

First-line treatment is oral ferrous sulfate 200 mg (65 mg elemental iron) three times daily for at least three months after hemoglobin correction to replenish iron stores 1, 2. Alternative formulations include ferrous gluconate or ferrous fumarate if ferrous sulfate is not tolerated 1. Adding ascorbic acid enhances iron absorption 1, 2.

Response Monitoring

• A good response is defined as hemoglobin rise ≥10 g/L within 2 weeks, confirming iron deficiency 1, 2
• Monitor hemoglobin and red cell indices at three-monthly intervals for one year, then after a further year 1, 2
• For non-responders to oral iron, consider intravenous iron if malabsorption is present, expecting hemoglobin increase of at least 2 g/dL within 4 weeks 1, 2

Special Considerations

• Genetic disorders affecting iron metabolism (SLC11A2 defects) may require oral iron supplementation and/or erythropoietin and/or erythrocyte transfusions 1
• X-linked sideroblastic anemia (ALAS2 defects) should be treated with pyridoxine 50-200 mg daily initially, with lifelong maintenance at 10-100 mg daily 1
• STEAP3 defects require erythrocyte transfusions combined with erythropoietin, with chelation for systemic iron loading 1

Immune Thrombocytopenia (ITP)

Newly Diagnosed ITP

Use a short course of prednisone (≤6 weeks including taper) rather than prolonged courses (>6 weeks) 3. For children with non-life-threatening mucosal bleeding, corticosteroids are preferred over anti-D immunoglobulin 3.

Persistent/Chronic ITP (≥3 months)

For corticosteroid-dependent or unresponsive patients, thrombopoietin receptor agonists (eltrombopag or romiplostim) are suggested as second-line therapy 3.

The treatment hierarchy for second-line therapy depends on disease duration and patient priorities 3:

• For ITP <12 months duration: TPO-RAs are preferred over rituximab due to greater durability of response 3
• For ITP >12 months duration:
  • Patients avoiding surgery: TPO-RAs preferred over rituximab 3
  • Patients avoiding long-term medication: Rituximab preferred over splenectomy 3
  • Patients seeking durable response: Either TPO-RAs or splenectomy 3

Pediatric Considerations

Children with platelet count <20 × 10⁹/L and only mild bleeding (skin manifestations) should be managed as outpatients rather than admitted 3. This also applies to children with platelet count ≥20 × 10⁹/L with mild bleeding 3.

Hemochromatosis

Therapeutic phlebotomy is the first-line treatment for iron depletion in hemochromatosis 3. Erythrocytapheresis is an alternative requiring fewer interventions and can be cost-effective during the induction phase 3.

Treatment Protocol

• Induction phase: Weekly or biweekly phlebotomy until ferritin reaches 50 μg/L 3
• Maintenance phase: Continue phlebotomy to maintain ferritin 50-100 μg/L 3
• Check hemoglobin before each phlebotomy; reduce rate if <12 g/dL, pause if <11 g/dL 3
• Check ferritin after 4 phlebotomies 3

Critical Counseling Points

• Avoid iron and vitamin C supplements, daily red meat consumption, and moderate to heavy alcohol intake 3
• Patients with advanced liver disease must abstain from alcohol completely 3
• Avoid raw/undercooked seafood and contact of wounds with seawater due to Vibrio vulnificus infection risk 3

Chemotherapy-Associated Anemia

Before initiating erythropoiesis-stimulating agents (ESAs), evaluate and correct other causes of anemia including iron, folate, and B12 deficiency, occult blood loss, and renal insufficiency 3.

ESA Therapy

• Use epoetin or darbepoetin when hemoglobin approaches or falls below 10 g/dL 3
• These agents are considered equivalent in effectiveness and safety 3
• RBC transfusion remains an option depending on severity of anemia or clinical circumstances 3

Thromboembolism Risk

Carefully weigh thromboembolism risks before prescribing ESAs 3. Randomized trials demonstrate increased thromboembolism risk in patients receiving epoetin or darbepoetin 3. Multiple myeloma patients receiving thalidomide or lenalidomide with doxorubicin or corticosteroids are at particularly increased risk 3.

Chronic Myelomonocytic Leukemia (CMML)

Myelodysplastic Phenotype (MD-CMML)

• Patients with <10% blasts: Manage with supportive therapy correcting cytopenias 3
• Severe anemia (Hb ≤10 g/dL) with erythropoietin ≤500 mU/dL: Treat with erythropoietic stimulating agents 3
• Patients with ≥10% blasts in bone marrow or ≥5% in blood: Integrate supportive therapy with hypomethylating agents (5-azacytidine or decitabine) 3

Myeloproliferative Phenotype (MP-CMML)

• Low blast count: Hydroxyurea is the drug of choice to control proliferative cells and reduce organomegaly 3
• High blast count: Polychemotherapy followed by allogeneic stem cell transplantation when possible 3

Second-Line Options

For patients resistant or intolerant to hydroxyurea without blasts, cytolytic therapy with VP16, low-dose ARA-C, or thioguanine as single agents 3. Hypomethylating agents should be used in clinical trials 3.

Sickle Cell Disease: Transfusion Complications

For delayed hemolytic transfusion reactions with ongoing hyperhemolysis, initiate immunosuppressive therapy promptly 3.

Immunosuppressive Protocol

• First-line agents: IVIg (0.4-1 g/kg per day for 3-5 days, up to 2 g/kg total) and high-dose steroids (methylprednisolone or prednisone 1-4 mg/kg per day) 3
• Second-line agent: Eculizumab 3
• Rituximab: Primarily indicated for preventing additional alloantibody formation (375 mg/m² repeated after 2 weeks) 3

Critical Management Points

• Avoid further transfusion unless life-threatening anemia with ongoing hemolysis 3
• If transfusion warranted, use extended matched red cells (C/c, E/e, K, Jka/Jkb, Fya/Fyb, S/s) 3
• Initiate supportive care including erythropoietin with or without IV iron 3

Chronic Myelogenous Leukemia (CML)

Supportive Care for Cytopenias

For symptomatic leukocytosis: Hydroxyurea, apheresis, imatinib, or clinical trial 3.

For symptomatic thrombocytosis: Hydroxyurea, anti-aggregants, anagrelide, or apheresis 3.

Imatinib Dose Modifications

• Grade 3-4 neutropenia (ANC <1000/mm³): Hold until ANC ≥1500/mm³, resume at 400 mg; if recurrent, resume at 300 mg 3
• Grade 3-4 thrombocytopenia (platelets <50,000/mm³): Hold until platelets ≥75,000/mm³, resume at 400 mg; if recurrent, resume at 300 mg 3
• Growth factors can be used in combination with imatinib for resistant neutropenia 3

Allogeneic Stem Cell Transplantation

Allogeneic stem cell transplantation is the only curative strategy for CMML 3. HLA typing is recommended for patients aged under 65 years 3. For SLC25A38 defects causing sideroblastic anemia, hematopoietic stem cell transplantation is the only curative option 1.