Orphan drugs for the treatment of hairy cell leukemia

Figures & data

Figure 1. May Grunwald Giemsa stained peripheral blood film from a patient with HCL showing circulating hairy cells.

Table 1. Laboratory features of HCL and other splenomegalic B-cell disorders with villous lymphocytes.

	HCL	HCL-variant	DRSBCL	SMZL
Nucleus	Kidney shaped	Round	Round/oval	Round/oval
Nucleolus	No	Yes	No	No
Cytoplasm	Abundant	Abundant	Medium/small	Small
	Villous	Villous	Villous basophilia	Villous basophilia
Spleen	Red pulp pseudolakes	Red pulp pseudolakes +/−	Red pulp	marginal zone
Bone marrow*	Interstitial/diffuse spacing	Interstitial or diffuse spacing	Interstitial nodular	Interstitial nodular
CD11c	+++	+++	+/++	+/++
CD25	+++	neg	+	+
CD103	+++	++	+	neg
CD123	+++	neg	neg	neg
TRAP	+++	neg	NA	neg
Cyclin D1	+++	neg	NA	neg

*Intrasinusoidal infiltration may be present in all diseases.

+++: Positive in > 60% cases; ++: Positive in 30 – 60% cases; +: Positive in 10 – 30% of cases; neg: Positive in < 10% of cases; DRPSBCL: Diffuse red pulp splenic B-cell lymphoma; HCL: Hairy cell leukemia; HCL-v: Hairy cell leukemia variant; NA: Not available; SMZL: Splenic marginal zone lymphoma.

Figure 2. Diagnostic work up of a patient with HCL.

Table 2. Progress of treatments in HCL.

Agent	Ref.	Year
Splenectomy		1970s
Interferon-α	[35]	1984
2-deoxycoformycin	[43]	1984
2-chorodeoxyadenosine	[44]	1990
Immuntoxin conjugates	[73-83]	2000s

Table 3. Responses to deoxycoformycin in HCL.

No of patients	Prior therapy*	ORR (%)	CR (%)	PR (%)	Ref.
15	15	93	93	0	[45]
23	13	91	87	4	[46]
28	18	100	89	11	[47]
33	30	78	33	45	[48]
49	31	96	44	52	[49]
50	31	84	64	20	[50]
66	66	79	56	23	[51]
80	43	98	72	16	[30]
85	85	84	42	42	[52]
148	23	96	74	22	[53]
154	0	79	76	3	[25]
188^‡	108	96	82	14	[24]
238	154	95.6	79	16.6	[28]
240	86	72	NA	NA	[29]

*Including splenectomy and/or Interferon and/or others.

^‡Patients in this series were also included in reference 53.

CR: Complete response; ORR: Overall response rate; PR: Partial response.

Table 4. Responses to CDA in HCL.

No of patients	Prior therapy*	ORR (%)	CR (%)	PR (%)	Ref.
97	41	95.9	77.3	18.6	[54]
29	14	96	86	10	[55]
37	12	100	78	22	[56]
46	27	89	78	11	[57]
44	11	98	98	0	[58]
48	18	100	76	24	[24]
49	28	100	76	24	[59]
50	15	100	80	20	[60]
144	75	97	85	12	[61]
196	na	100	95	5	[27]
349	176	98	91	7	[31]
861	513	87	50	37	[26]

*Including splenectomy and/or Interferon and/or others.

CR: Complete response; ORR: Overall response rate; PR: Partial response.

Figure 3. Flow chart illustrating the treatment pathways in HCL.

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