The modern management of severe aplastic anaemia

Ball, Sarah E.

doi:10.1046/j.1365-2141.2000.02081.x

Cited by 31 publications

(32 citation statements)

References 104 publications

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“…[2][3][4] BMT from a phenotypically HLA-matched unrelated donor is indicated as salvage therapy for patients who fail to respond to one or more courses of IST and patients who experience relapse of disease. 5,6 Initial attempts to use unrelated donor BMT for patients with SAA were hampered by the high incidence of graft failure and graft versus host disease (GVHD); survival rates ranged from 20% to 54%. [7][8][9][10] An optimum conditioning regimen, GVHD prophylaxis, and better donor selection are considered to be indispensable for improving outcomes for patients who receive unrelated donor BMT.…”

Section: Introductionmentioning

confidence: 99%

Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program

Kojima¹,

Matsuyama²,

Kato³

et al. 2002

Blood

162

143

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Section: Introductionmentioning

confidence: 99%

Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program

Kojima¹,

Matsuyama²,

Kato³

et al. 2002

Blood

162

143

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“…Der Grund hierfür liegt in einer Optimierung und Erweiterung der Behandlungsmög-lichkeiten, die sich in supportive und kausale Therapieverfahren einteilen lassen (2). Zur Planung der Behandlungsstrategie sollte jeder Patient mit einer aplastischen Anämie zum Diagnosezeitpunkt und zur Durchführung kausaler Therapien in einem hämato-logischen Zentrum vorgestellt werden.…”

Section: Aaunclassified

“…Hierdurch wird per se eine Verbesserung der Krankheitsprognose erzielt; außerdem werden durch supportive Verfahren die klinischen Voraussetzungen für eine ursächliche Behandlung geschaffen (2 …”

Section: Supportive Therapieunclassified

Aplastic anemia - Treatment

Schroers

Hohloch²,

Trumper³

2003

Dtsch med Wochenschr

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Glossar AA= aplastische Anämie ALG = Anti-Lymphozyten-Globulin ATG = Anti-Thymozyten-Globulin CsA = Ciclosporin A GvHD = Gaft-versus-Host-Erkrankung IST = Immunsuppressive Therapie KMT = Knochenmarktransplantation PNH = Paroxysmale nächtliche Hämoglobinurie SAA = schwere aplastische Anämie VSAA = sehr (very) schwere aplastische Anämie Die Prognose erworbener aplastischer Anämien hat sich in den vergangenen zwei Jahrzehnten stetig verbessert. Der Grund hierfür liegt in einer Optimierung und Erweiterung der Behandlungsmög-lichkeiten, die sich in supportive und kausale Therapieverfahren einteilen lassen (2). Zur Planung der Behandlungsstrategie sollte jeder Patient mit einer aplastischen Anämie zum Diagnosezeitpunkt und zur Durchführung kausaler Therapien in einem hämato-logischen Zentrum vorgestellt werden. Grundsätzlich orientiert sich die individuelle Therapie am Schweregrad der Erkrankung, dem Alter und klinischen Zustand des Patienten sowie der Verfüg-barkeit eines HLA-identischen Familienspenders für eine allogene Knochenmarktransplantation (11). Supportive Therapie

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“…Allogeneic BMT from a human leukemic antigen (HLA)-matched sibling donor provides curative therapy for patients with AA but is limited by donor availability. [1][2][3][4][5][6] Immunosuppression with antithymocyte globulin (ATG) and cyclosporine A is an effective alternative treatment. 7,8 Hematologic improvement generally occurs within 3 months, and up to 70% of patients respond.…”

mentioning

confidence: 99%

Sequential interleukin 3 and granulocyte‐macrophage–colony stimulating factor therapy in patients with bone marrow failure with long‐term follow‐up of responses

Talpaz

Champlin

et al. 2003

Cancer

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BACKGROUNDInterleukin‐3 (IL‐3) and granulocyte‐macrophage–colony stimulating factor (GM‐CSF) have synergistic, hematopoietic growth‐promoting activity in preclinical studies. Because of the paucity of effective therapies for patients with chronic bone marrow failure states, the authors studied the biologic activity of sequential IL‐3/GM‐CSF in such patients.METHODSIL‐3 was given subcutaneously for 5 days (at escalating doses of 0.15 μg/kg, 0.3 μg/kg, 0.6 μg/kg, 1.2 μg/kg, 2.5 μg/kg, 5.0 μg/kg, 10.0 μg/kg, or 15.0 μg/kg per day), and GM‐CSF for was given subcutaneously for 9 days (at a dose of 5 μg/kg per day; Phase I 3 + 3 design) followed by 14 days of rest (total, 2 courses), then maintenance therapy.RESULTSThe majority of 38 evaluable patients had aplastic anemia or myelodysplastic syndrome. Most patients (79%) had neutrophil responses. Ten patients (26%), all of whom were treated with IL‐3 doses ≥ 1.2 μg/kg per day, had platelet responses, with a median increase of 132 × 109/L (range, 41–180 × 109/L) over baseline in responders. Six patients (16%) had trilineage recovery, which could be durable (the longest ongoing at 6.5 years after therapy completion). The most common toxicities were low‐grade fever, headache, and fatigue. The maximum tolerated doses were IL‐3 at 10 μg/kg per day and GM‐CSF at 5 μg/kg per day.CONCLUSIONSSequential IL‐3/GM‐CSF effectively raised blood counts in some patients with bone marrow failure at doses that were tolerated well. These results indicate that early‐acting growth factors can induce durable, multilineage responses in a subset of individuals with bone marrow failure. Cancer 2003. © 2003 American Cancer Society.

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The modern management of severe aplastic anaemia

Cited by 31 publications

References 104 publications

Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program

Outcome of 154 patients with severe aplastic anemia who received transplants from unrelated donors: the Japan Marrow Donor Program

Aplastic anemia - Treatment

Sequential interleukin 3 and granulocyte‐macrophage–colony stimulating factor therapy in patients with bone marrow failure with long‐term follow‐up of responses

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