Phase II Pilot Study of Granulocyte Colony-Stimulating Factor for Inherited Bone Marrow Failure Syndromes

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

1994-12-31

Brief Summary

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OBJECTIVES: I. Assess the efficacy of recombinant human granulocyte colony-stimulating factor (G-CSF) in raising the absolute neutrophil count, platelet count, and hemoglobin level in patients with inherited bone marrow failure syndromes.

II. Assess the efficacy of a reduced maintenance dose in patients who respond to daily G-CSF.

III. Assess the toxic effects of G-CSF in these patients. IV. Measure bone marrow progenitor colonies before and after G-CSF. V. Measure CD34-positive cells in marrow and blood before and after G-CSF using flow cytometry and immunohistochemistry.

Detailed Description

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PROTOCOL OUTLINE: Patients receive granulocyte colony-stimulating factor (G-CSF) subcutaneously every day for 8 weeks; nonresponders receive an increased dose for an additional 8 weeks. Patients who respond at week 8 or 16 are then tapered to a lower maintenance dose of G-CSF administered every other day through week 40. The dose is adjusted to maintain an absolute neutrophil count above 1500.

Patients are removed from study for failure to achieve a complete response by week 16, unacceptable nonhematologic toxicity, the identification of a clonal karyotype in marrow, or the onset of leukemia.

Conditions

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Shwachman Syndrome Fanconi's Anemia Dyskeratosis Congenita Thrombocytopenia

Keywords

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Fanconi's anemia Shwachman syndrome aplastic anemia dermatologic disorders dyskeratosis congenita hematologic disorders rare disease thrombocytopenia

Study Design

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Primary Study Purpose

TREATMENT

Interventions

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filgrastim

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Inherited bone marrow failure syndrome, including:

* Fanconi's anemia
* Dyskeratosis congenita
* Shwachman syndrome
* Amegakaryocytic thrombocytopenia
* Decreased megakaryocytes in infancy
* No thrombocytopenia with absent radius syndrome (TAR)
* No trisomy 13 or 18
* No clonal bone marrow karyotype

--Prior/Concurrent Therapy--

* At least 4 weeks since growth factors
* Concurrent therapy allowed if not altered for 30 days prior to entry through week 8
* No concurrent investigational drugs

--Patient Characteristics--

* Hematopoietic: ANC \<1000
* No leukemia
* Other: No medical or psychiatric contraindication to protocol participation
* No pregnant or nursing women

Minimum Eligible Age

0 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Principal Investigators

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David A. Williams

Role: STUDY_CHAIR

James Whitcomb Riley Hospital for Children

References

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Rackoff WR, Orazi A, Robinson CA, Cooper RJ, Alter BP, Freedman MH, Harris RE, Williams DA. Prolonged administration of granulocyte colony-stimulating factor (filgrastim) to patients with Fanconi anemia: a pilot study. Blood. 1996 Sep 1;88(5):1588-93.

Reference Type BACKGROUND

PMID: 8781414 (View on PubMed)

Other Identifiers

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UTMB-416

Identifier Type: -

Identifier Source: secondary_id

199/11877